FDA/CDC

 

The Food and Drug Administration is concerned about incidents of overheating, fires, and explosions of e-cigarettes, or “vapes,” which in some cases have resulted in serious injuries. The agency is reviewing these types of incidents and has taken steps to protect the public.

The FDA recently developed resources for consumers, including tips to help avoid e-cigarette overheating and explosions, as well as social media tools to help spread the word about protective steps. The agency also has a reporting system to collect information about adverse experiences associated with e-cigarettes and other tobacco products. Comprehensive and accurate reports could provide evidence to help inform future actions to protect the public.

The FDA has provided tips that may help reduce the risk of e-cigarette overheating and explosions. The agency’s Center for Tobacco Products (CTP) encourages you to share these tips with patients who use or might use these tobacco products, as well as with health professionals, parents, and others who might pass along the tips.
 

For e-cigarette users: Tips to help prevent fires and explosions

Learn about your device.

The best protection against battery explosions may be knowing about your device and how to handle and charge its batteries appropriately. Read and follow the manufacturer’s use and care recommendations. If the e-cigarette did not come with instructions or you have additional questions, contact the manufacturer.

Consider using e-cigarettes with protective features.

Some e-cigarettes have features such as firing button locks, vent holes, and protection against overcharging. These features are designed to prevent battery overheating and explosions, so do not remove or disable them.

Choose batteries carefully and replace them if necessary:

• Use only the batteries recommended for your device. Do not mix different brands, different charge levels, or old and new batteries in the same device.

• Replace the batteries if they get damaged or wet. If your e-cigarette is damaged and the batteries are not replaceable, contact the manufacturer.

• Stop using the device under certain circumstances. Although battery explosions can occur with no warning, you should immediately stop using your e-cigarette and get a safe distance from it if you notice any of these during use or while charging: strange noises; unusual smells; a leaking battery; the e-cigarette becoming unusually hot; or the device beginning to smoke, spark, emit flashes, or catch fire.

Be aware when charging your e-cigarette:

• Use only the charger that came with your device, and never charge it with a phone or tablet charger.

• Charge the device on a clean, flat surface, in a place you can clearly see it, and away from anything that can easily catch fire. Do not leave the e-cigarette charging on a surface such as a couch or pillow, where it may be more likely to overheat or turn on unintentionally.

• Do not charge the device overnight or leave it charging unattended.

Know this about carrying and storing your e-cigarette:

• Keep your e-cigarette covered. If you are carrying it in your pocket, avoid having it come in contact with coins or loose batteries.

• Protect the e-cigarette from extreme temperatures. Do not leave it in direct sunlight or in your car in extremely cold or hot temperatures.

Report any problems.

If something goes wrong with an e-cigarette, please submit a report to https://www.safetyreporting.hhs.gov

To make it easy to share the FDA’s top tips, the agency has developed a 5 Tips to Help Avoid “Vape” Battery Explosion infographic.

This infographic and other public-health resources can be found on a dedicated CTP webpage, which offers shareable and downloadable content to help spread the word about e-cigarette battery issues, as well as a video on how to report adverse experiences related to tobacco products to the FDA.
 

When a fire or explosion does occur: The Safety Reporting Portal

The CTP identified 143 reported incidents of e-cigarette overheating, fires, and explosions during 2009-2015, and 20 additional reports during 2016. Based on the FDA’s experience with underreporting of adverse events for other regulated products, the number of actual events is probably higher.

The FDA is working to collect more information to identify the true number of events and why these incidents are occurring. The agency has a Safety Reporting Portal (SRP) dedicated to receiving reports of issues associated with FDA-regulated products, including e-cigarettes and other tobacco products.

The FDA strongly encourages any physicians, other health care professionals, or those with firsthand knowledge about an unexpected e-cigarette incident to report it through the SRP. Family physicians can play a valuable reporting role by informing patients about the reporting system, helping people submit complete information about incidents related to e-cigarettes, or providing information about an incident on a patient’s behalf.

To report an e-cigarette failure or other tobacco-related adverse event, please go to the SRP and follow the instructions in each section. Those unable to use the SRP to submit a report can call 877-CTP-1373 or email AskCTP@fda.hhs.gov.

The more complete and accurate a report is, the more helpful it can be to the FDA and, in turn, to public health. When submitting a report about an e-cigarette, please include:

• E-cigarette manufacturer’s name.

• E-cigarette’s brand name, model, and serial number.

• Battery’s brand name and model.

• Place the e-cigarette was purchased.

• Whether, and how, the product was being used at the time of the incident.

• Whether the product was used differently than intended by the manufacturer.

• Whether the product was modified in any way.

To collect as much detail as possible, the FDA encourages those submitting reports to upload photos or other files, such as police or hospital reports. They also appreciate submission of contact information, such as a phone number or email address, which will help the agency follow up with any questions related to the report. Personal information will not be shared or used for any additional matter and is protected by security practices. The HHS Privacy Policy contains more information.
 

Ongoing public health protection efforts

The FDA continues to evaluate possible ways to protect the public from device-related fires and explosions. During a public workshop in April, the FDA heard from experts including scientists, engineers, and e-cigarette manufacturers and retailers, as well as from the general public, about hazards and possible solutions related to batteries in e-cigarettes and other electronic nicotine delivery systems. Also, the agency’s premarket review process for electronic nicotine delivery systems includes an assessment of device operation and any features that may reduce the risks associated with product use, including testing related to overheating and exploding batteries.

Through these and other measures, the FDA is committed to identifying and addressing factors leading to e-cigarette overheating and any subsequent injuries. Health care professionals can help by spreading the word about the agency’s user tips and reporting portal.

To learn more broadly about the FDA’s ongoing efforts to protect the public health by regulating the manufacture, marketing, and distribution of tobacco products, please visit the Center for Tobacco Products website.

Dr. Holman is director of the office of science at the FDA’s Center for Tobacco Products.

 

The Food and Drug Administration is concerned about incidents of overheating, fires, and explosions of e-cigarettes, or “vapes,” which in some cases have resulted in serious injuries. The agency is reviewing these types of incidents and has taken steps to protect the public.

The FDA recently developed resources for consumers, including tips to help avoid e-cigarette overheating and explosions, as well as social media tools to help spread the word about protective steps. The agency also has a reporting system to collect information about adverse experiences associated with e-cigarettes and other tobacco products. Comprehensive and accurate reports could provide evidence to help inform future actions to protect the public.

The FDA has provided tips that may help reduce the risk of e-cigarette overheating and explosions. The agency’s Center for Tobacco Products (CTP) encourages you to share these tips with patients who use or might use these tobacco products, as well as with health professionals, parents, and others who might pass along the tips.
 

For e-cigarette users: Tips to help prevent fires and explosions

Learn about your device.

The best protection against battery explosions may be knowing about your device and how to handle and charge its batteries appropriately. Read and follow the manufacturer’s use and care recommendations. If the e-cigarette did not come with instructions or you have additional questions, contact the manufacturer.

Consider using e-cigarettes with protective features.

Some e-cigarettes have features such as firing button locks, vent holes, and protection against overcharging. These features are designed to prevent battery overheating and explosions, so do not remove or disable them.

Choose batteries carefully and replace them if necessary:

• Use only the batteries recommended for your device. Do not mix different brands, different charge levels, or old and new batteries in the same device.

• Replace the batteries if they get damaged or wet. If your e-cigarette is damaged and the batteries are not replaceable, contact the manufacturer.

• Stop using the device under certain circumstances. Although battery explosions can occur with no warning, you should immediately stop using your e-cigarette and get a safe distance from it if you notice any of these during use or while charging: strange noises; unusual smells; a leaking battery; the e-cigarette becoming unusually hot; or the device beginning to smoke, spark, emit flashes, or catch fire.

Be aware when charging your e-cigarette:

• Use only the charger that came with your device, and never charge it with a phone or tablet charger.

• Charge the device on a clean, flat surface, in a place you can clearly see it, and away from anything that can easily catch fire. Do not leave the e-cigarette charging on a surface such as a couch or pillow, where it may be more likely to overheat or turn on unintentionally.

• Do not charge the device overnight or leave it charging unattended.

Know this about carrying and storing your e-cigarette:

• Keep your e-cigarette covered. If you are carrying it in your pocket, avoid having it come in contact with coins or loose batteries.

• Protect the e-cigarette from extreme temperatures. Do not leave it in direct sunlight or in your car in extremely cold or hot temperatures.

Report any problems.

If something goes wrong with an e-cigarette, please submit a report to https://www.safetyreporting.hhs.gov

To make it easy to share the FDA’s top tips, the agency has developed a 5 Tips to Help Avoid “Vape” Battery Explosion infographic.

This infographic and other public-health resources can be found on a dedicated CTP webpage, which offers shareable and downloadable content to help spread the word about e-cigarette battery issues, as well as a video on how to report adverse experiences related to tobacco products to the FDA.
 

When a fire or explosion does occur: The Safety Reporting Portal

The CTP identified 143 reported incidents of e-cigarette overheating, fires, and explosions during 2009-2015, and 20 additional reports during 2016. Based on the FDA’s experience with underreporting of adverse events for other regulated products, the number of actual events is probably higher.

The FDA is working to collect more information to identify the true number of events and why these incidents are occurring. The agency has a Safety Reporting Portal (SRP) dedicated to receiving reports of issues associated with FDA-regulated products, including e-cigarettes and other tobacco products.

The FDA strongly encourages any physicians, other health care professionals, or those with firsthand knowledge about an unexpected e-cigarette incident to report it through the SRP. Family physicians can play a valuable reporting role by informing patients about the reporting system, helping people submit complete information about incidents related to e-cigarettes, or providing information about an incident on a patient’s behalf.

To report an e-cigarette failure or other tobacco-related adverse event, please go to the SRP and follow the instructions in each section. Those unable to use the SRP to submit a report can call 877-CTP-1373 or email AskCTP@fda.hhs.gov.

The more complete and accurate a report is, the more helpful it can be to the FDA and, in turn, to public health. When submitting a report about an e-cigarette, please include:

• E-cigarette manufacturer’s name.

• E-cigarette’s brand name, model, and serial number.

• Battery’s brand name and model.

• Place the e-cigarette was purchased.

• Whether, and how, the product was being used at the time of the incident.

• Whether the product was used differently than intended by the manufacturer.

• Whether the product was modified in any way.

To collect as much detail as possible, the FDA encourages those submitting reports to upload photos or other files, such as police or hospital reports. They also appreciate submission of contact information, such as a phone number or email address, which will help the agency follow up with any questions related to the report. Personal information will not be shared or used for any additional matter and is protected by security practices. The HHS Privacy Policy contains more information.
 

Ongoing public health protection efforts

The FDA continues to evaluate possible ways to protect the public from device-related fires and explosions. During a public workshop in April, the FDA heard from experts including scientists, engineers, and e-cigarette manufacturers and retailers, as well as from the general public, about hazards and possible solutions related to batteries in e-cigarettes and other electronic nicotine delivery systems. Also, the agency’s premarket review process for electronic nicotine delivery systems includes an assessment of device operation and any features that may reduce the risks associated with product use, including testing related to overheating and exploding batteries.

Through these and other measures, the FDA is committed to identifying and addressing factors leading to e-cigarette overheating and any subsequent injuries. Health care professionals can help by spreading the word about the agency’s user tips and reporting portal.

To learn more broadly about the FDA’s ongoing efforts to protect the public health by regulating the manufacture, marketing, and distribution of tobacco products, please visit the Center for Tobacco Products website.

Dr. Holman is director of the office of science at the FDA’s Center for Tobacco Products.

 

The Food and Drug Administration is concerned about incidents of overheating, fires, and explosions of e-cigarettes, or “vapes,” which in some cases have resulted in serious injuries. The agency is reviewing these types of incidents and has taken steps to protect the public.

The FDA recently developed resources for consumers, including tips to help avoid e-cigarette overheating and explosions, as well as social media tools to help spread the word about protective steps. The agency also has a reporting system to collect information about adverse experiences associated with e-cigarettes and other tobacco products. Comprehensive and accurate reports could provide evidence to help inform future actions to protect the public.

The FDA has provided tips that may help reduce the risk of e-cigarette overheating and explosions. The agency’s Center for Tobacco Products (CTP) encourages you to share these tips with patients who use or might use these tobacco products, as well as with health professionals, parents, and others who might pass along the tips.
 

For e-cigarette users: Tips to help prevent fires and explosions

Learn about your device.

The best protection against battery explosions may be knowing about your device and how to handle and charge its batteries appropriately. Read and follow the manufacturer’s use and care recommendations. If the e-cigarette did not come with instructions or you have additional questions, contact the manufacturer.

Consider using e-cigarettes with protective features.

Some e-cigarettes have features such as firing button locks, vent holes, and protection against overcharging. These features are designed to prevent battery overheating and explosions, so do not remove or disable them.

Choose batteries carefully and replace them if necessary:

• Use only the batteries recommended for your device. Do not mix different brands, different charge levels, or old and new batteries in the same device.

• Replace the batteries if they get damaged or wet. If your e-cigarette is damaged and the batteries are not replaceable, contact the manufacturer.

• Stop using the device under certain circumstances. Although battery explosions can occur with no warning, you should immediately stop using your e-cigarette and get a safe distance from it if you notice any of these during use or while charging: strange noises; unusual smells; a leaking battery; the e-cigarette becoming unusually hot; or the device beginning to smoke, spark, emit flashes, or catch fire.

Be aware when charging your e-cigarette:

• Use only the charger that came with your device, and never charge it with a phone or tablet charger.

• Charge the device on a clean, flat surface, in a place you can clearly see it, and away from anything that can easily catch fire. Do not leave the e-cigarette charging on a surface such as a couch or pillow, where it may be more likely to overheat or turn on unintentionally.

• Do not charge the device overnight or leave it charging unattended.

Know this about carrying and storing your e-cigarette:

• Keep your e-cigarette covered. If you are carrying it in your pocket, avoid having it come in contact with coins or loose batteries.

• Protect the e-cigarette from extreme temperatures. Do not leave it in direct sunlight or in your car in extremely cold or hot temperatures.

Report any problems.

If something goes wrong with an e-cigarette, please submit a report to https://www.safetyreporting.hhs.gov

To make it easy to share the FDA’s top tips, the agency has developed a 5 Tips to Help Avoid “Vape” Battery Explosion infographic.

This infographic and other public-health resources can be found on a dedicated CTP webpage, which offers shareable and downloadable content to help spread the word about e-cigarette battery issues, as well as a video on how to report adverse experiences related to tobacco products to the FDA.
 

When a fire or explosion does occur: The Safety Reporting Portal

The CTP identified 143 reported incidents of e-cigarette overheating, fires, and explosions during 2009-2015, and 20 additional reports during 2016. Based on the FDA’s experience with underreporting of adverse events for other regulated products, the number of actual events is probably higher.

The FDA is working to collect more information to identify the true number of events and why these incidents are occurring. The agency has a Safety Reporting Portal (SRP) dedicated to receiving reports of issues associated with FDA-regulated products, including e-cigarettes and other tobacco products.

The FDA strongly encourages any physicians, other health care professionals, or those with firsthand knowledge about an unexpected e-cigarette incident to report it through the SRP. Family physicians can play a valuable reporting role by informing patients about the reporting system, helping people submit complete information about incidents related to e-cigarettes, or providing information about an incident on a patient’s behalf.

To report an e-cigarette failure or other tobacco-related adverse event, please go to the SRP and follow the instructions in each section. Those unable to use the SRP to submit a report can call 877-CTP-1373 or email AskCTP@fda.hhs.gov.

The more complete and accurate a report is, the more helpful it can be to the FDA and, in turn, to public health. When submitting a report about an e-cigarette, please include:

• E-cigarette manufacturer’s name.

• E-cigarette’s brand name, model, and serial number.

• Battery’s brand name and model.

• Place the e-cigarette was purchased.

• Whether, and how, the product was being used at the time of the incident.

• Whether the product was used differently than intended by the manufacturer.

• Whether the product was modified in any way.

To collect as much detail as possible, the FDA encourages those submitting reports to upload photos or other files, such as police or hospital reports. They also appreciate submission of contact information, such as a phone number or email address, which will help the agency follow up with any questions related to the report. Personal information will not be shared or used for any additional matter and is protected by security practices. The HHS Privacy Policy contains more information.
 

Ongoing public health protection efforts

The FDA continues to evaluate possible ways to protect the public from device-related fires and explosions. During a public workshop in April, the FDA heard from experts including scientists, engineers, and e-cigarette manufacturers and retailers, as well as from the general public, about hazards and possible solutions related to batteries in e-cigarettes and other electronic nicotine delivery systems. Also, the agency’s premarket review process for electronic nicotine delivery systems includes an assessment of device operation and any features that may reduce the risks associated with product use, including testing related to overheating and exploding batteries.

Through these and other measures, the FDA is committed to identifying and addressing factors leading to e-cigarette overheating and any subsequent injuries. Health care professionals can help by spreading the word about the agency’s user tips and reporting portal.

To learn more broadly about the FDA’s ongoing efforts to protect the public health by regulating the manufacture, marketing, and distribution of tobacco products, please visit the Center for Tobacco Products website.

Dr. Holman is director of the office of science at the FDA’s Center for Tobacco Products.

The Food and Drug Administration has approved the FreeStyle Libre Flash Glucose Monitoring System for the continuous monitoring of glucose in adults with diabetes, the first system of its type that does not require blood samples for calibration, according to a press release.

Instead of using a more standard finger stick with which patients must draw blood samples multiple times a day to measure glucose levels, the FreeStyle Libre Flash Glucose Monitoring System uses a thin wire less than 0.4-mm thick inserted underneath the skin in the back of the upper arm to continually monitor glucose. Blood glucose levels are read by swiping a mobile reader over the wire. After a 12-hour start-up period, the wire can be worn for 10 days.

FDA approval was based on results from a study of diabetes patients older than 18 years, as well as a performance review comparing readings obtained by the device with readings obtained in a traditional laboratory method utilizing blood samples.

“This system allows people with diabetes to avoid the additional step of finger-stick calibration, which can sometimes be painful, but still provides necessary information for treating their diabetes – with a wave of the mobile reader,” Donald St. Pierre, acting director of the Office of In Vitro Diagnostics and Radiological Health and deputy director of new product evaluation in the FDA’s Center for Devices and Radiological Health, said in the press release.

Find the full press release on the FDA website.

 

lfranki@frontlinemedcom.com

The Food and Drug Administration has approved the FreeStyle Libre Flash Glucose Monitoring System for the continuous monitoring of glucose in adults with diabetes, the first system of its type that does not require blood samples for calibration, according to a press release.

Instead of using a more standard finger stick with which patients must draw blood samples multiple times a day to measure glucose levels, the FreeStyle Libre Flash Glucose Monitoring System uses a thin wire less than 0.4-mm thick inserted underneath the skin in the back of the upper arm to continually monitor glucose. Blood glucose levels are read by swiping a mobile reader over the wire. After a 12-hour start-up period, the wire can be worn for 10 days.

FDA approval was based on results from a study of diabetes patients older than 18 years, as well as a performance review comparing readings obtained by the device with readings obtained in a traditional laboratory method utilizing blood samples.

“This system allows people with diabetes to avoid the additional step of finger-stick calibration, which can sometimes be painful, but still provides necessary information for treating their diabetes – with a wave of the mobile reader,” Donald St. Pierre, acting director of the Office of In Vitro Diagnostics and Radiological Health and deputy director of new product evaluation in the FDA’s Center for Devices and Radiological Health, said in the press release.

Find the full press release on the FDA website.

 

lfranki@frontlinemedcom.com

The Food and Drug Administration has approved the FreeStyle Libre Flash Glucose Monitoring System for the continuous monitoring of glucose in adults with diabetes, the first system of its type that does not require blood samples for calibration, according to a press release.

Instead of using a more standard finger stick with which patients must draw blood samples multiple times a day to measure glucose levels, the FreeStyle Libre Flash Glucose Monitoring System uses a thin wire less than 0.4-mm thick inserted underneath the skin in the back of the upper arm to continually monitor glucose. Blood glucose levels are read by swiping a mobile reader over the wire. After a 12-hour start-up period, the wire can be worn for 10 days.

FDA approval was based on results from a study of diabetes patients older than 18 years, as well as a performance review comparing readings obtained by the device with readings obtained in a traditional laboratory method utilizing blood samples.

“This system allows people with diabetes to avoid the additional step of finger-stick calibration, which can sometimes be painful, but still provides necessary information for treating their diabetes – with a wave of the mobile reader,” Donald St. Pierre, acting director of the Office of In Vitro Diagnostics and Radiological Health and deputy director of new product evaluation in the FDA’s Center for Devices and Radiological Health, said in the press release.

Find the full press release on the FDA website.

 

lfranki@frontlinemedcom.com

The Food and Drug Administration has approved pembrolizumab (Keytruda) for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma, in cases where tests confirm that the tumors contain programmed death–ligand 1 and where the disease is progressing on or after two or more prior lines of therapy.

Pembrolizumab has been approved in the United States since 2014 for the treatment of melanoma, with subsequent approvals for treatment of non–small-cell lung cancer, head and neck squamous cell carcinoma, Hodgkin lymphoma, and several other advanced cancers.

The drug, now approved under the FDA’s accelerated approval regulations for the current indication in a 50-mg and 100-mg injection, blocks the interaction between the PD-1 protein and its ligands.

The approval comes on the basis of the nonrandomized, open label KEYNOTE-059 trial, which enrolled 259 patients with gastric or GEJ adenocarcinoma that progressed on at least two prior systemic treatments for advanced disease. Of the enrollees, 143 patients had tumors with a PD-L1 Combined Positive Score of 1 or greater. The primary trial outcome, the objective response rate for these 143 patients, was 13.3% (95% confidence interval; 8.2-20), with a complete response rate of 1.4% and a partial response rate of 11.9%. The duration of response ranged from at least 2.8 months to at least 19.4 months.

Continued approval for the new indication will depend upon further demonstration of a clinical benefit in confirmatory trials.

dwatson@frontlinemedcom.com

The Food and Drug Administration has approved pembrolizumab (Keytruda) for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma, in cases where tests confirm that the tumors contain programmed death–ligand 1 and where the disease is progressing on or after two or more prior lines of therapy.

Pembrolizumab has been approved in the United States since 2014 for the treatment of melanoma, with subsequent approvals for treatment of non–small-cell lung cancer, head and neck squamous cell carcinoma, Hodgkin lymphoma, and several other advanced cancers.

The drug, now approved under the FDA’s accelerated approval regulations for the current indication in a 50-mg and 100-mg injection, blocks the interaction between the PD-1 protein and its ligands.

The approval comes on the basis of the nonrandomized, open label KEYNOTE-059 trial, which enrolled 259 patients with gastric or GEJ adenocarcinoma that progressed on at least two prior systemic treatments for advanced disease. Of the enrollees, 143 patients had tumors with a PD-L1 Combined Positive Score of 1 or greater. The primary trial outcome, the objective response rate for these 143 patients, was 13.3% (95% confidence interval; 8.2-20), with a complete response rate of 1.4% and a partial response rate of 11.9%. The duration of response ranged from at least 2.8 months to at least 19.4 months.

Continued approval for the new indication will depend upon further demonstration of a clinical benefit in confirmatory trials.

dwatson@frontlinemedcom.com

The Food and Drug Administration has approved pembrolizumab (Keytruda) for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma, in cases where tests confirm that the tumors contain programmed death–ligand 1 and where the disease is progressing on or after two or more prior lines of therapy.

Pembrolizumab has been approved in the United States since 2014 for the treatment of melanoma, with subsequent approvals for treatment of non–small-cell lung cancer, head and neck squamous cell carcinoma, Hodgkin lymphoma, and several other advanced cancers.

The drug, now approved under the FDA’s accelerated approval regulations for the current indication in a 50-mg and 100-mg injection, blocks the interaction between the PD-1 protein and its ligands.

The approval comes on the basis of the nonrandomized, open label KEYNOTE-059 trial, which enrolled 259 patients with gastric or GEJ adenocarcinoma that progressed on at least two prior systemic treatments for advanced disease. Of the enrollees, 143 patients had tumors with a PD-L1 Combined Positive Score of 1 or greater. The primary trial outcome, the objective response rate for these 143 patients, was 13.3% (95% confidence interval; 8.2-20), with a complete response rate of 1.4% and a partial response rate of 11.9%. The duration of response ranged from at least 2.8 months to at least 19.4 months.

Continued approval for the new indication will depend upon further demonstration of a clinical benefit in confirmatory trials.

dwatson@frontlinemedcom.com

 

The Food and Drug Administration has granted accelerated approval to nivolumab (Optivo) for the treatment of patients with hepatocellular carcinoma (HCC) who have been treated previously with sorafenib.

Approval was based on tumor response rate and durability of response from the phase 1/2 Checkmate 040 trial. Of 154 patients with HCC in the trial who received nivolumab following progression on sorafenib, 3 experienced complete response, and 19 experienced partial response. Median time to response was 2.8 months, and 91% of patients who responded to treatment had a response length longer than 6 months, with 55% of patients having a response length longer than 1 year.

Courtesy Wikimedia Commons/FitzColinGerald/Creative Commons License

The most common side effects associated with nivolumab include fatigue, musculoskeletal pain, abdominal pain, pruritus, diarrhea, rash, cough, and decreased appetite. Nivolumab can cause immune-mediated pneumonitis, colitis, hepatitis, endocrinopathies, nephritis and renal dysfunction, adverse skin reactions, encephalitis, as well as infusion reactions and embryo-fetal toxicity, according to a press release from Bristol-Myers Squibb announcing the approval.

“In recent years, there has been growing interest in leveraging immuno-oncology knowledge and discoveries to add to the treatment options available for patients with advanced-stage liver cancer. The approval of Opdivo provides us with an encouraging approach and a new treatment option for appropriate patients with HCC following prior systemic therapy,” Anthony B. El-Khoueiry, MD, a medical oncologist and phase 1 program director at the University of Southern California, Los Angeles, and the USC Norris Comprehensive Cancer Center, said in the press release.

lfranki@frontlinemedcom.com

 

The Food and Drug Administration has granted accelerated approval to nivolumab (Optivo) for the treatment of patients with hepatocellular carcinoma (HCC) who have been treated previously with sorafenib.

Approval was based on tumor response rate and durability of response from the phase 1/2 Checkmate 040 trial. Of 154 patients with HCC in the trial who received nivolumab following progression on sorafenib, 3 experienced complete response, and 19 experienced partial response. Median time to response was 2.8 months, and 91% of patients who responded to treatment had a response length longer than 6 months, with 55% of patients having a response length longer than 1 year.

Courtesy Wikimedia Commons/FitzColinGerald/Creative Commons License

The most common side effects associated with nivolumab include fatigue, musculoskeletal pain, abdominal pain, pruritus, diarrhea, rash, cough, and decreased appetite. Nivolumab can cause immune-mediated pneumonitis, colitis, hepatitis, endocrinopathies, nephritis and renal dysfunction, adverse skin reactions, encephalitis, as well as infusion reactions and embryo-fetal toxicity, according to a press release from Bristol-Myers Squibb announcing the approval.

“In recent years, there has been growing interest in leveraging immuno-oncology knowledge and discoveries to add to the treatment options available for patients with advanced-stage liver cancer. The approval of Opdivo provides us with an encouraging approach and a new treatment option for appropriate patients with HCC following prior systemic therapy,” Anthony B. El-Khoueiry, MD, a medical oncologist and phase 1 program director at the University of Southern California, Los Angeles, and the USC Norris Comprehensive Cancer Center, said in the press release.

lfranki@frontlinemedcom.com

 

The Food and Drug Administration has granted accelerated approval to nivolumab (Optivo) for the treatment of patients with hepatocellular carcinoma (HCC) who have been treated previously with sorafenib.

Approval was based on tumor response rate and durability of response from the phase 1/2 Checkmate 040 trial. Of 154 patients with HCC in the trial who received nivolumab following progression on sorafenib, 3 experienced complete response, and 19 experienced partial response. Median time to response was 2.8 months, and 91% of patients who responded to treatment had a response length longer than 6 months, with 55% of patients having a response length longer than 1 year.

Courtesy Wikimedia Commons/FitzColinGerald/Creative Commons License

The most common side effects associated with nivolumab include fatigue, musculoskeletal pain, abdominal pain, pruritus, diarrhea, rash, cough, and decreased appetite. Nivolumab can cause immune-mediated pneumonitis, colitis, hepatitis, endocrinopathies, nephritis and renal dysfunction, adverse skin reactions, encephalitis, as well as infusion reactions and embryo-fetal toxicity, according to a press release from Bristol-Myers Squibb announcing the approval.

“In recent years, there has been growing interest in leveraging immuno-oncology knowledge and discoveries to add to the treatment options available for patients with advanced-stage liver cancer. The approval of Opdivo provides us with an encouraging approach and a new treatment option for appropriate patients with HCC following prior systemic therapy,” Anthony B. El-Khoueiry, MD, a medical oncologist and phase 1 program director at the University of Southern California, Los Angeles, and the USC Norris Comprehensive Cancer Center, said in the press release.

lfranki@frontlinemedcom.com

Aegerion Pharmaceuticals has agreed to plead guilty in the United States District Court for the District of Massachusetts to two misdemeanor counts of violating the Federal Food, Drug, and Cosmetic Act (FD&C Act) involving the introduction of misbranded Juxtapid (lomitapide) into interstate commerce, according to a press release from the Food and Drug Administration.

Juxtapid was misbranded because Aegerion failed to comply with the requirements of the Juxtapid Risk Evaluation and Mitigation Strategy (REMS) program and because the drug’s labeling lacked adequate directions for all of Juxtapid’s intended uses, according to the charges. Aegerion also agreed to a comprehensive compliance program and legal tools for the FDA to ensure that Aegerion complies with the law, subject to judicial oversight.

“By failing to follow the safety requirements that Aegerion had agreed to, the company put patients’ lives at risk and didn’t honor the safety commitments they made as a condition of gaining approval for their drug. This is unacceptable. We will continue to pursue those who skirt the law, and flout patient safety and other postmarket commitments, using all of the enforcement tools available to us. Postmarket safety requirements are a key element of the FDA’s public health protections and we will ensure that they are fulfilled,” FDA Commissioner Scott Gottlieb, MD, said in the statement.

Rather than following the REMS requirement to distribute Juxtapid only for the narrow indication of homozygous familial hypercholesterolemia, Aegerion portrayed the definition of the rare disorder as vague and indefinite in order to extend its use to lower-risk patients. Further, Aegerion filed a misleading REMS assessment report to the FDA in which the company failed to disclose that it was distributing Juxtapid using this definition, which was inconsistent with Aegerion’s preapproval filings and peer-reviewed clinical standards of diagnosis, according to the FDA release.

Once entered by the court, the plea and consent decree will be part of a global resolution of multiple government investigations into Aegerion’s conduct with respect to the marketing and distribution of Juxtapid. This resolution was the result of a coordinated effort by the U.S. Department of Justice and several government agencies, including the FDA, the press release stated.

Juxtapid was approved in December 2012 as an adjunct therapy to treat homozygous familial hypercholesterolemia. The Juxtapid REMS requires Aegerion to educate prescribers about the risks of hepatotoxicity and the need to monitor patients treated with Juxtapid and to ensure that Juxtapid is prescribed and dispensed only to those patients with a clinical or laboratory diagnosis consistent with homozygous familial hypercholesterolemia.

Aegerion Pharmaceuticals has agreed to plead guilty in the United States District Court for the District of Massachusetts to two misdemeanor counts of violating the Federal Food, Drug, and Cosmetic Act (FD&C Act) involving the introduction of misbranded Juxtapid (lomitapide) into interstate commerce, according to a press release from the Food and Drug Administration.

Juxtapid was misbranded because Aegerion failed to comply with the requirements of the Juxtapid Risk Evaluation and Mitigation Strategy (REMS) program and because the drug’s labeling lacked adequate directions for all of Juxtapid’s intended uses, according to the charges. Aegerion also agreed to a comprehensive compliance program and legal tools for the FDA to ensure that Aegerion complies with the law, subject to judicial oversight.

“By failing to follow the safety requirements that Aegerion had agreed to, the company put patients’ lives at risk and didn’t honor the safety commitments they made as a condition of gaining approval for their drug. This is unacceptable. We will continue to pursue those who skirt the law, and flout patient safety and other postmarket commitments, using all of the enforcement tools available to us. Postmarket safety requirements are a key element of the FDA’s public health protections and we will ensure that they are fulfilled,” FDA Commissioner Scott Gottlieb, MD, said in the statement.

Rather than following the REMS requirement to distribute Juxtapid only for the narrow indication of homozygous familial hypercholesterolemia, Aegerion portrayed the definition of the rare disorder as vague and indefinite in order to extend its use to lower-risk patients. Further, Aegerion filed a misleading REMS assessment report to the FDA in which the company failed to disclose that it was distributing Juxtapid using this definition, which was inconsistent with Aegerion’s preapproval filings and peer-reviewed clinical standards of diagnosis, according to the FDA release.

Once entered by the court, the plea and consent decree will be part of a global resolution of multiple government investigations into Aegerion’s conduct with respect to the marketing and distribution of Juxtapid. This resolution was the result of a coordinated effort by the U.S. Department of Justice and several government agencies, including the FDA, the press release stated.

Juxtapid was approved in December 2012 as an adjunct therapy to treat homozygous familial hypercholesterolemia. The Juxtapid REMS requires Aegerion to educate prescribers about the risks of hepatotoxicity and the need to monitor patients treated with Juxtapid and to ensure that Juxtapid is prescribed and dispensed only to those patients with a clinical or laboratory diagnosis consistent with homozygous familial hypercholesterolemia.

Aegerion Pharmaceuticals has agreed to plead guilty in the United States District Court for the District of Massachusetts to two misdemeanor counts of violating the Federal Food, Drug, and Cosmetic Act (FD&C Act) involving the introduction of misbranded Juxtapid (lomitapide) into interstate commerce, according to a press release from the Food and Drug Administration.

Juxtapid was misbranded because Aegerion failed to comply with the requirements of the Juxtapid Risk Evaluation and Mitigation Strategy (REMS) program and because the drug’s labeling lacked adequate directions for all of Juxtapid’s intended uses, according to the charges. Aegerion also agreed to a comprehensive compliance program and legal tools for the FDA to ensure that Aegerion complies with the law, subject to judicial oversight.

“By failing to follow the safety requirements that Aegerion had agreed to, the company put patients’ lives at risk and didn’t honor the safety commitments they made as a condition of gaining approval for their drug. This is unacceptable. We will continue to pursue those who skirt the law, and flout patient safety and other postmarket commitments, using all of the enforcement tools available to us. Postmarket safety requirements are a key element of the FDA’s public health protections and we will ensure that they are fulfilled,” FDA Commissioner Scott Gottlieb, MD, said in the statement.

Rather than following the REMS requirement to distribute Juxtapid only for the narrow indication of homozygous familial hypercholesterolemia, Aegerion portrayed the definition of the rare disorder as vague and indefinite in order to extend its use to lower-risk patients. Further, Aegerion filed a misleading REMS assessment report to the FDA in which the company failed to disclose that it was distributing Juxtapid using this definition, which was inconsistent with Aegerion’s preapproval filings and peer-reviewed clinical standards of diagnosis, according to the FDA release.

Once entered by the court, the plea and consent decree will be part of a global resolution of multiple government investigations into Aegerion’s conduct with respect to the marketing and distribution of Juxtapid. This resolution was the result of a coordinated effort by the U.S. Department of Justice and several government agencies, including the FDA, the press release stated.

Juxtapid was approved in December 2012 as an adjunct therapy to treat homozygous familial hypercholesterolemia. The Juxtapid REMS requires Aegerion to educate prescribers about the risks of hepatotoxicity and the need to monitor patients treated with Juxtapid and to ensure that Juxtapid is prescribed and dispensed only to those patients with a clinical or laboratory diagnosis consistent with homozygous familial hypercholesterolemia.

The Food and Drug Administration advised on Sept. 21 that incorrect excessive dosing of the liver disease medicine obeticholic acid (Ocaliva) creates an increased risk of serious liver injury and death. The agency recommends closer monitoring of dosages and following the current label’s recommendations.

Obeticholic acid is used to treat primary biliary cholangitis. Patients taking the drug should first be tested for their baseline liver function so that the effects of the drug can be monitored. The level of liver impairment determines the recommended dosage: Those with moderate to severe impairment should start by taking 5 mg weekly (with a possible increase to 10 mg weekly) instead of the standard 5 mg daily. Those who start at the standard dose and progress to moderate or severe liver impairment can have their dosage reduced or can discontinue the drug altogether.

 

dwatson@frontlinemedcom.com

The Food and Drug Administration advised on Sept. 21 that incorrect excessive dosing of the liver disease medicine obeticholic acid (Ocaliva) creates an increased risk of serious liver injury and death. The agency recommends closer monitoring of dosages and following the current label’s recommendations.

Obeticholic acid is used to treat primary biliary cholangitis. Patients taking the drug should first be tested for their baseline liver function so that the effects of the drug can be monitored. The level of liver impairment determines the recommended dosage: Those with moderate to severe impairment should start by taking 5 mg weekly (with a possible increase to 10 mg weekly) instead of the standard 5 mg daily. Those who start at the standard dose and progress to moderate or severe liver impairment can have their dosage reduced or can discontinue the drug altogether.

 

dwatson@frontlinemedcom.com

The Food and Drug Administration advised on Sept. 21 that incorrect excessive dosing of the liver disease medicine obeticholic acid (Ocaliva) creates an increased risk of serious liver injury and death. The agency recommends closer monitoring of dosages and following the current label’s recommendations.

Obeticholic acid is used to treat primary biliary cholangitis. Patients taking the drug should first be tested for their baseline liver function so that the effects of the drug can be monitored. The level of liver impairment determines the recommended dosage: Those with moderate to severe impairment should start by taking 5 mg weekly (with a possible increase to 10 mg weekly) instead of the standard 5 mg daily. Those who start at the standard dose and progress to moderate or severe liver impairment can have their dosage reduced or can discontinue the drug altogether.

 

dwatson@frontlinemedcom.com

 

The opioid addiction medications buprenorphine and methadone should not be withheld from patients who are taking benzodiazepines or other drugs that depress the central nervous system, the Food and Drug Administration advised in a safety alert posted Sept. 20.

The combined use of these medication-assisted treatment (MAT) drugs and central nervous system (CNS) depressants can lead to serious side effects. But the harm associated with opioid addiction that remains untreated usually outweighs those risks, the agency said. After reviewing this issue, the FDA said, it is requiring that this information be added to the drug labels of buprenorphine and methadone in addition to “recommendations for minimizing the use of [MAT] drugs and benzodiazepines together.”

The agency’s safety alert encouraged health professionals to develop a treatment plan for patients who are taking MAT drugs in combination with CNS depressants. The recommendations included educating patients about the risks of combined use of these drugs, “including overdose and death”; considering other treatment options for patients taking benzodiazepines for anxiety or insomnia; monitoring patients for illicit drug use, “including urine or blood screening”; communicating with other prescribers so that they know about patients’ buprenorphine or methadone treatment; and trying to taper the use of the benzodiazepine or CNS depressant to eventual discontinuation if possible.

Discontinuing MAT drugs is a goal, but patients might need medication-assisted treatment indefinitely. “Use should continue for as long as patients are benefiting and their use contributes to the intended treatment goals,” the alert said.

dwatson@frontlinemedcom.com

 

The opioid addiction medications buprenorphine and methadone should not be withheld from patients who are taking benzodiazepines or other drugs that depress the central nervous system, the Food and Drug Administration advised in a safety alert posted Sept. 20.

The combined use of these medication-assisted treatment (MAT) drugs and central nervous system (CNS) depressants can lead to serious side effects. But the harm associated with opioid addiction that remains untreated usually outweighs those risks, the agency said. After reviewing this issue, the FDA said, it is requiring that this information be added to the drug labels of buprenorphine and methadone in addition to “recommendations for minimizing the use of [MAT] drugs and benzodiazepines together.”

The agency’s safety alert encouraged health professionals to develop a treatment plan for patients who are taking MAT drugs in combination with CNS depressants. The recommendations included educating patients about the risks of combined use of these drugs, “including overdose and death”; considering other treatment options for patients taking benzodiazepines for anxiety or insomnia; monitoring patients for illicit drug use, “including urine or blood screening”; communicating with other prescribers so that they know about patients’ buprenorphine or methadone treatment; and trying to taper the use of the benzodiazepine or CNS depressant to eventual discontinuation if possible.

Discontinuing MAT drugs is a goal, but patients might need medication-assisted treatment indefinitely. “Use should continue for as long as patients are benefiting and their use contributes to the intended treatment goals,” the alert said.

dwatson@frontlinemedcom.com

 

The opioid addiction medications buprenorphine and methadone should not be withheld from patients who are taking benzodiazepines or other drugs that depress the central nervous system, the Food and Drug Administration advised in a safety alert posted Sept. 20.

The combined use of these medication-assisted treatment (MAT) drugs and central nervous system (CNS) depressants can lead to serious side effects. But the harm associated with opioid addiction that remains untreated usually outweighs those risks, the agency said. After reviewing this issue, the FDA said, it is requiring that this information be added to the drug labels of buprenorphine and methadone in addition to “recommendations for minimizing the use of [MAT] drugs and benzodiazepines together.”

The agency’s safety alert encouraged health professionals to develop a treatment plan for patients who are taking MAT drugs in combination with CNS depressants. The recommendations included educating patients about the risks of combined use of these drugs, “including overdose and death”; considering other treatment options for patients taking benzodiazepines for anxiety or insomnia; monitoring patients for illicit drug use, “including urine or blood screening”; communicating with other prescribers so that they know about patients’ buprenorphine or methadone treatment; and trying to taper the use of the benzodiazepine or CNS depressant to eventual discontinuation if possible.

Discontinuing MAT drugs is a goal, but patients might need medication-assisted treatment indefinitely. “Use should continue for as long as patients are benefiting and their use contributes to the intended treatment goals,” the alert said.

dwatson@frontlinemedcom.com

The Food and Drug Administration has approved the Pentax ED34-i10T model duodenoscope, the first with a disposable distal cap, according to an FDA press release.

A disposable distal cap will improve the ability to clean and reprocess the duodenoscope. Without being thoroughly cleaned and disinfected, contaminated tissue can remain and potentially can be transmitted to other patients.

“We believe the new disposable distal cap represents a major step toward lowering the risk of future infections associated with these devices,” William Maisel, MD, acting director of the Office of Device Evaluation at the FDA Center for Devices and Radiological Health, said in a statement. “Improving the safety of duodenoscopes is a top priority for the FDA, and we encourage companies to continue to pursue innovations that will help reduce the risk to patients.”

A previous version of the Pentax duodenoscope, the ED-3490TK, was subject to a January 2017 FDA Safety Alert, because of the potential for cracks and gaps to develop in the adhesive sealing the duodenoscope’s distal cap.

lfranki@frontlinemedcom.com

The Food and Drug Administration has approved the Pentax ED34-i10T model duodenoscope, the first with a disposable distal cap, according to an FDA press release.

A disposable distal cap will improve the ability to clean and reprocess the duodenoscope. Without being thoroughly cleaned and disinfected, contaminated tissue can remain and potentially can be transmitted to other patients.

“We believe the new disposable distal cap represents a major step toward lowering the risk of future infections associated with these devices,” William Maisel, MD, acting director of the Office of Device Evaluation at the FDA Center for Devices and Radiological Health, said in a statement. “Improving the safety of duodenoscopes is a top priority for the FDA, and we encourage companies to continue to pursue innovations that will help reduce the risk to patients.”

A previous version of the Pentax duodenoscope, the ED-3490TK, was subject to a January 2017 FDA Safety Alert, because of the potential for cracks and gaps to develop in the adhesive sealing the duodenoscope’s distal cap.

lfranki@frontlinemedcom.com

The Food and Drug Administration has approved the Pentax ED34-i10T model duodenoscope, the first with a disposable distal cap, according to an FDA press release.

A disposable distal cap will improve the ability to clean and reprocess the duodenoscope. Without being thoroughly cleaned and disinfected, contaminated tissue can remain and potentially can be transmitted to other patients.

“We believe the new disposable distal cap represents a major step toward lowering the risk of future infections associated with these devices,” William Maisel, MD, acting director of the Office of Device Evaluation at the FDA Center for Devices and Radiological Health, said in a statement. “Improving the safety of duodenoscopes is a top priority for the FDA, and we encourage companies to continue to pursue innovations that will help reduce the risk to patients.”

A previous version of the Pentax duodenoscope, the ED-3490TK, was subject to a January 2017 FDA Safety Alert, because of the potential for cracks and gaps to develop in the adhesive sealing the duodenoscope’s distal cap.

lfranki@frontlinemedcom.com

 

The Food and Drug Administration has granted accelerated approval to copanlisib (Aliqopa) for the treatment of adults with relapsed follicular lymphoma who have received at least two prior treatments.

Approval of the kinase inhibitor was based on an overall response rate of 59% in a single-arm trial of 104 patients with follicular B-cell non-Hodgkin lymphoma who had relapsed disease following at least two prior treatments. These patients had a complete or partial response for a median 12.2 months.

Previously, copanlisib was granted priority review and orphan drug designation. Common side effects included hyperglycemia, diarrhea, decreased general strength and energy, hypertension, leukopenia, neutropenia, nausea, lower respiratory tract infections, and thrombocytopenia, the FDA said in a press release.

“For patients with relapsed follicular lymphoma, the cancer often comes back even after multiple treatments,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research said in the press release. “Options are limited for these patients and today’s approval provides an additional choice for treatment, filling an unmet need for them,” he said.

llaubach@frontlinemedcom.com

 

The Food and Drug Administration has granted accelerated approval to copanlisib (Aliqopa) for the treatment of adults with relapsed follicular lymphoma who have received at least two prior treatments.

Approval of the kinase inhibitor was based on an overall response rate of 59% in a single-arm trial of 104 patients with follicular B-cell non-Hodgkin lymphoma who had relapsed disease following at least two prior treatments. These patients had a complete or partial response for a median 12.2 months.

Previously, copanlisib was granted priority review and orphan drug designation. Common side effects included hyperglycemia, diarrhea, decreased general strength and energy, hypertension, leukopenia, neutropenia, nausea, lower respiratory tract infections, and thrombocytopenia, the FDA said in a press release.

“For patients with relapsed follicular lymphoma, the cancer often comes back even after multiple treatments,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research said in the press release. “Options are limited for these patients and today’s approval provides an additional choice for treatment, filling an unmet need for them,” he said.

llaubach@frontlinemedcom.com

 

The Food and Drug Administration has granted accelerated approval to copanlisib (Aliqopa) for the treatment of adults with relapsed follicular lymphoma who have received at least two prior treatments.

Approval of the kinase inhibitor was based on an overall response rate of 59% in a single-arm trial of 104 patients with follicular B-cell non-Hodgkin lymphoma who had relapsed disease following at least two prior treatments. These patients had a complete or partial response for a median 12.2 months.

Previously, copanlisib was granted priority review and orphan drug designation. Common side effects included hyperglycemia, diarrhea, decreased general strength and energy, hypertension, leukopenia, neutropenia, nausea, lower respiratory tract infections, and thrombocytopenia, the FDA said in a press release.

“For patients with relapsed follicular lymphoma, the cancer often comes back even after multiple treatments,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research said in the press release. “Options are limited for these patients and today’s approval provides an additional choice for treatment, filling an unmet need for them,” he said.

llaubach@frontlinemedcom.com

 

SILVER SPRING, MD. – Alopecia areata patients struggle as much, if not more so, with the social and emotional challenges of the disease as with the physical challenges, according to patients and others who spoke at a public meeting on alopecia areata patient-focused drug development.

Alopecia areata affects as many as 6.8 million individuals in the United States, according to the National Alopecia Areata Foundation (NAAF). However, the particulars of alopecia can vary widely from one person to another; some patients experience total hair loss (alopecia universalis), while others retain eyebrows, eyelashes, or some body hair.

The FDA meeting, held on Sept. 11, is part of the agency’s patient-focused drug development initiative. “We wanted to hear the broader patient’s voice,” Theresa M. Mullin, PhD, director of the FDA’s Office of Strategic Programs, said in her opening remarks. Gary Sherwood, communications director for NAAF, said that the meeting was the culmination of a 5-year effort, begun in 2012 when alopecia areata was named as one of 39 disease categories under consideration for such a meeting. “It is too early to know what the exact results will be … but if the past is any indication, they may be significant. The meeting held with psoriasis yielded FDA approval of a treatment previously denied,” he added in an interview.

Two panel presentations featured patients who discussed their experiences with alopecia; each was followed by a discussion period where patients and family members in the audience were invited to share their experiences.

The “Health Effects and Daily Impacts” panel allowed several patients and their family members the opportunity to identify specific issues that may surprise clinicians.

Heidi Splete/Frontline Medical News

“I found this session to be very moving. Many of the patient stories brought me to tears,” Kalyani Marathe, MD, a dermatologist at Children’s National Health System, Washington, said in an interview. “Being exposed to the raw pain that they felt inspired me to take more time to listen to my own patients’ stories and to ask more questions about their condition and the impact it has on their lives.

“One thing I learned was how much the patients are bothered by sweating of the scalp; this can affect what type of head covering, hair piece, or hat/helmet they are able to wear, and thus limits activities,” Dr. Marathe continued. “This is not something I had focused on previously. I will be more inclined to ask about sweating and offer treatments, such as scalp botulinum toxin or aluminum chloride now that I have been alerted to this concern. Also, the challenges of facial makeup such as pencil for eyebrows was another thing that the FDA session brought home for me; I’m more inclined to suggest things such as microblading for eyebrows, or to try treatments like latanoprost for eyebrows/lashes.”

The second panel, “Current Approaches to Treatment,” included a different group of patients who shared stories of treatments that had been successful and those that had not. “The patients at the FDA meeting expressed very eloquently what our patients feel – different treatments may work temporarily and then stop working, which leads to a roller coaster of emotions of hope and disappointment,” A. Yasmine Kirkorian, MD, also a dermatologist at Children’s National Health System, said in an interview. “Patients and physicians would be interested in a treatment option with a track record for predictable efficacy with durable and sustained hair regrowth and minimal side effects.”

Dr. Marathe noted that in her experience, those who develop alopecia totalis or universalis at a younger age tend to have more recalcitrant disease. “It is still very hard for me to predict which children will regrow their hair spontaneously, or with topical therapies, versus those with more resistant disease. I hope that continued study will allow us to offer a more realistic prognosis for these patients,” she said.

Discussion after the treatment panel included testimonials from patients who reported successful treatment with tofacitinib (Xeljanz), a Janus kinase inhibitor approved for rheumatoid arthritis, which is not approved for treatment of alopecia.

“I absolutely agree with the focus on JAK inhibitors and increasing our understanding of how they work, as well as what some of the long-term effects are,” said Dr. Marathe. “The better we are able to target the pathogenesis of this condition, the more easily we can treat in a more focused fashion and reduce side effects,” but more clinical trials are needed to determine safety and efficacy for children and teens, she noted.

One of her hesitations in prescribing tofacitinib to her patients is that she cannot provide them with a sense of how long they will need to be on the treatment. “Current data show that the hair growth on the medication is usually lost upon stopping it; the question I still struggle with is whether it is realistic to put a 4- or 5-year-old on a medication that has no estimated or anticipated stop date,” she said.

As for what she offers patients in terms of resources for emotional support, Dr. Kirkorian said the psychosocial aspects of alopecia areata are always discussed at patient visits. “Psychosocial needs vary based on age, personality, and personal philosophy. We offer the gamut of outside resources from local support groups, the National Alopecia Areata Foundation, referral to psychology/psychiatry and, very importantly, referral to Camp Discovery. Children have told us across the board how important and meaningful it was to them to be able to just be themselves around other children who look like them.”

Dr. Marathe and Dr. Kirkorian were attendees at the meeting; they had no relevant disclosures. They are members of the Dermatology News Editorial Advisory Board.

 

SILVER SPRING, MD. – Alopecia areata patients struggle as much, if not more so, with the social and emotional challenges of the disease as with the physical challenges, according to patients and others who spoke at a public meeting on alopecia areata patient-focused drug development.

Alopecia areata affects as many as 6.8 million individuals in the United States, according to the National Alopecia Areata Foundation (NAAF). However, the particulars of alopecia can vary widely from one person to another; some patients experience total hair loss (alopecia universalis), while others retain eyebrows, eyelashes, or some body hair.

The FDA meeting, held on Sept. 11, is part of the agency’s patient-focused drug development initiative. “We wanted to hear the broader patient’s voice,” Theresa M. Mullin, PhD, director of the FDA’s Office of Strategic Programs, said in her opening remarks. Gary Sherwood, communications director for NAAF, said that the meeting was the culmination of a 5-year effort, begun in 2012 when alopecia areata was named as one of 39 disease categories under consideration for such a meeting. “It is too early to know what the exact results will be … but if the past is any indication, they may be significant. The meeting held with psoriasis yielded FDA approval of a treatment previously denied,” he added in an interview.

Two panel presentations featured patients who discussed their experiences with alopecia; each was followed by a discussion period where patients and family members in the audience were invited to share their experiences.

The “Health Effects and Daily Impacts” panel allowed several patients and their family members the opportunity to identify specific issues that may surprise clinicians.

Heidi Splete/Frontline Medical News

“I found this session to be very moving. Many of the patient stories brought me to tears,” Kalyani Marathe, MD, a dermatologist at Children’s National Health System, Washington, said in an interview. “Being exposed to the raw pain that they felt inspired me to take more time to listen to my own patients’ stories and to ask more questions about their condition and the impact it has on their lives.

“One thing I learned was how much the patients are bothered by sweating of the scalp; this can affect what type of head covering, hair piece, or hat/helmet they are able to wear, and thus limits activities,” Dr. Marathe continued. “This is not something I had focused on previously. I will be more inclined to ask about sweating and offer treatments, such as scalp botulinum toxin or aluminum chloride now that I have been alerted to this concern. Also, the challenges of facial makeup such as pencil for eyebrows was another thing that the FDA session brought home for me; I’m more inclined to suggest things such as microblading for eyebrows, or to try treatments like latanoprost for eyebrows/lashes.”

The second panel, “Current Approaches to Treatment,” included a different group of patients who shared stories of treatments that had been successful and those that had not. “The patients at the FDA meeting expressed very eloquently what our patients feel – different treatments may work temporarily and then stop working, which leads to a roller coaster of emotions of hope and disappointment,” A. Yasmine Kirkorian, MD, also a dermatologist at Children’s National Health System, said in an interview. “Patients and physicians would be interested in a treatment option with a track record for predictable efficacy with durable and sustained hair regrowth and minimal side effects.”

Dr. Marathe noted that in her experience, those who develop alopecia totalis or universalis at a younger age tend to have more recalcitrant disease. “It is still very hard for me to predict which children will regrow their hair spontaneously, or with topical therapies, versus those with more resistant disease. I hope that continued study will allow us to offer a more realistic prognosis for these patients,” she said.

Discussion after the treatment panel included testimonials from patients who reported successful treatment with tofacitinib (Xeljanz), a Janus kinase inhibitor approved for rheumatoid arthritis, which is not approved for treatment of alopecia.

“I absolutely agree with the focus on JAK inhibitors and increasing our understanding of how they work, as well as what some of the long-term effects are,” said Dr. Marathe. “The better we are able to target the pathogenesis of this condition, the more easily we can treat in a more focused fashion and reduce side effects,” but more clinical trials are needed to determine safety and efficacy for children and teens, she noted.

One of her hesitations in prescribing tofacitinib to her patients is that she cannot provide them with a sense of how long they will need to be on the treatment. “Current data show that the hair growth on the medication is usually lost upon stopping it; the question I still struggle with is whether it is realistic to put a 4- or 5-year-old on a medication that has no estimated or anticipated stop date,” she said.

As for what she offers patients in terms of resources for emotional support, Dr. Kirkorian said the psychosocial aspects of alopecia areata are always discussed at patient visits. “Psychosocial needs vary based on age, personality, and personal philosophy. We offer the gamut of outside resources from local support groups, the National Alopecia Areata Foundation, referral to psychology/psychiatry and, very importantly, referral to Camp Discovery. Children have told us across the board how important and meaningful it was to them to be able to just be themselves around other children who look like them.”

Dr. Marathe and Dr. Kirkorian were attendees at the meeting; they had no relevant disclosures. They are members of the Dermatology News Editorial Advisory Board.

 

SILVER SPRING, MD. – Alopecia areata patients struggle as much, if not more so, with the social and emotional challenges of the disease as with the physical challenges, according to patients and others who spoke at a public meeting on alopecia areata patient-focused drug development.

Alopecia areata affects as many as 6.8 million individuals in the United States, according to the National Alopecia Areata Foundation (NAAF). However, the particulars of alopecia can vary widely from one person to another; some patients experience total hair loss (alopecia universalis), while others retain eyebrows, eyelashes, or some body hair.

The FDA meeting, held on Sept. 11, is part of the agency’s patient-focused drug development initiative. “We wanted to hear the broader patient’s voice,” Theresa M. Mullin, PhD, director of the FDA’s Office of Strategic Programs, said in her opening remarks. Gary Sherwood, communications director for NAAF, said that the meeting was the culmination of a 5-year effort, begun in 2012 when alopecia areata was named as one of 39 disease categories under consideration for such a meeting. “It is too early to know what the exact results will be … but if the past is any indication, they may be significant. The meeting held with psoriasis yielded FDA approval of a treatment previously denied,” he added in an interview.

Two panel presentations featured patients who discussed their experiences with alopecia; each was followed by a discussion period where patients and family members in the audience were invited to share their experiences.

The “Health Effects and Daily Impacts” panel allowed several patients and their family members the opportunity to identify specific issues that may surprise clinicians.

Heidi Splete/Frontline Medical News

“I found this session to be very moving. Many of the patient stories brought me to tears,” Kalyani Marathe, MD, a dermatologist at Children’s National Health System, Washington, said in an interview. “Being exposed to the raw pain that they felt inspired me to take more time to listen to my own patients’ stories and to ask more questions about their condition and the impact it has on their lives.

“One thing I learned was how much the patients are bothered by sweating of the scalp; this can affect what type of head covering, hair piece, or hat/helmet they are able to wear, and thus limits activities,” Dr. Marathe continued. “This is not something I had focused on previously. I will be more inclined to ask about sweating and offer treatments, such as scalp botulinum toxin or aluminum chloride now that I have been alerted to this concern. Also, the challenges of facial makeup such as pencil for eyebrows was another thing that the FDA session brought home for me; I’m more inclined to suggest things such as microblading for eyebrows, or to try treatments like latanoprost for eyebrows/lashes.”

The second panel, “Current Approaches to Treatment,” included a different group of patients who shared stories of treatments that had been successful and those that had not. “The patients at the FDA meeting expressed very eloquently what our patients feel – different treatments may work temporarily and then stop working, which leads to a roller coaster of emotions of hope and disappointment,” A. Yasmine Kirkorian, MD, also a dermatologist at Children’s National Health System, said in an interview. “Patients and physicians would be interested in a treatment option with a track record for predictable efficacy with durable and sustained hair regrowth and minimal side effects.”

Dr. Marathe noted that in her experience, those who develop alopecia totalis or universalis at a younger age tend to have more recalcitrant disease. “It is still very hard for me to predict which children will regrow their hair spontaneously, or with topical therapies, versus those with more resistant disease. I hope that continued study will allow us to offer a more realistic prognosis for these patients,” she said.

Discussion after the treatment panel included testimonials from patients who reported successful treatment with tofacitinib (Xeljanz), a Janus kinase inhibitor approved for rheumatoid arthritis, which is not approved for treatment of alopecia.

“I absolutely agree with the focus on JAK inhibitors and increasing our understanding of how they work, as well as what some of the long-term effects are,” said Dr. Marathe. “The better we are able to target the pathogenesis of this condition, the more easily we can treat in a more focused fashion and reduce side effects,” but more clinical trials are needed to determine safety and efficacy for children and teens, she noted.

One of her hesitations in prescribing tofacitinib to her patients is that she cannot provide them with a sense of how long they will need to be on the treatment. “Current data show that the hair growth on the medication is usually lost upon stopping it; the question I still struggle with is whether it is realistic to put a 4- or 5-year-old on a medication that has no estimated or anticipated stop date,” she said.

As for what she offers patients in terms of resources for emotional support, Dr. Kirkorian said the psychosocial aspects of alopecia areata are always discussed at patient visits. “Psychosocial needs vary based on age, personality, and personal philosophy. We offer the gamut of outside resources from local support groups, the National Alopecia Areata Foundation, referral to psychology/psychiatry and, very importantly, referral to Camp Discovery. Children have told us across the board how important and meaningful it was to them to be able to just be themselves around other children who look like them.”

Dr. Marathe and Dr. Kirkorian were attendees at the meeting; they had no relevant disclosures. They are members of the Dermatology News Editorial Advisory Board.