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FDA: Cybersecurity vulnerabilities identified in GE Healthcare monitoring devices
The Food and Drug Administration has issued a warning that certain GE Healthcare Clinical Information Central Stations and Telemetry Servers have cybersecurity vulnerabilities that may introduce risk to monitored patients.
silence alarms, generate false alarms, and interfere with alarms of patient monitors connected to these devices, according to an “Urgent Medical Device Correction” letter issued by GE Healthcare in November 2019.
The affected devices are the ApexPro Telemetry Server and CARESCAPE Telemetry Server, the CARESCAPE Central Station (CSCS) version 1, and the CIC Pro Clinical Information Center Central Station version 1. These devices are used in health care facilities for displaying information, such as the patient’s physiological parameters, and for monitoring patient status from a central location in a facility.
No adverse events related to the vulnerabilities have been reported to the FDA. Health care facility staff should update their devices when GE Healthcare issues a software patch that addresses the vulnerability, separate the network connecting patient monitors using affected devices from the rest of the hospital, and use firewalls and other means to minimize the risk of remote or local network attacks.
“The FDA takes reports of cybersecurity vulnerabilities in medical devices seriously and will continue to work with GE Healthcare as the firm develops software patches to correct these vulnerabilities as soon as possible. The FDA will continue to assess new information concerning the vulnerabilities and will keep the public informed if significant new information becomes available,” the FDA said in the Safety Communication.
The Food and Drug Administration has issued a warning that certain GE Healthcare Clinical Information Central Stations and Telemetry Servers have cybersecurity vulnerabilities that may introduce risk to monitored patients.
silence alarms, generate false alarms, and interfere with alarms of patient monitors connected to these devices, according to an “Urgent Medical Device Correction” letter issued by GE Healthcare in November 2019.
The affected devices are the ApexPro Telemetry Server and CARESCAPE Telemetry Server, the CARESCAPE Central Station (CSCS) version 1, and the CIC Pro Clinical Information Center Central Station version 1. These devices are used in health care facilities for displaying information, such as the patient’s physiological parameters, and for monitoring patient status from a central location in a facility.
No adverse events related to the vulnerabilities have been reported to the FDA. Health care facility staff should update their devices when GE Healthcare issues a software patch that addresses the vulnerability, separate the network connecting patient monitors using affected devices from the rest of the hospital, and use firewalls and other means to minimize the risk of remote or local network attacks.
“The FDA takes reports of cybersecurity vulnerabilities in medical devices seriously and will continue to work with GE Healthcare as the firm develops software patches to correct these vulnerabilities as soon as possible. The FDA will continue to assess new information concerning the vulnerabilities and will keep the public informed if significant new information becomes available,” the FDA said in the Safety Communication.
The Food and Drug Administration has issued a warning that certain GE Healthcare Clinical Information Central Stations and Telemetry Servers have cybersecurity vulnerabilities that may introduce risk to monitored patients.
silence alarms, generate false alarms, and interfere with alarms of patient monitors connected to these devices, according to an “Urgent Medical Device Correction” letter issued by GE Healthcare in November 2019.
The affected devices are the ApexPro Telemetry Server and CARESCAPE Telemetry Server, the CARESCAPE Central Station (CSCS) version 1, and the CIC Pro Clinical Information Center Central Station version 1. These devices are used in health care facilities for displaying information, such as the patient’s physiological parameters, and for monitoring patient status from a central location in a facility.
No adverse events related to the vulnerabilities have been reported to the FDA. Health care facility staff should update their devices when GE Healthcare issues a software patch that addresses the vulnerability, separate the network connecting patient monitors using affected devices from the rest of the hospital, and use firewalls and other means to minimize the risk of remote or local network attacks.
“The FDA takes reports of cybersecurity vulnerabilities in medical devices seriously and will continue to work with GE Healthcare as the firm develops software patches to correct these vulnerabilities as soon as possible. The FDA will continue to assess new information concerning the vulnerabilities and will keep the public informed if significant new information becomes available,” the FDA said in the Safety Communication.
New Vascepa indication opens up treatment to millions; “Most significant event since statins”
The newly approved U.S. indication for icosapent ethyl (Vascepa; Amarin) is broadly in line with the entry criteria for the REDUCE-IT trial and includes a large high-risk primary-prevention population, as well as those with established cardiovascular disease (CVD). The drug, thus, could well be used by millions of patients in the United States alone.
The high-dose, purified eicosapentaenoic acid product was approved last week by the Food and Drug Administration for cardiovascular risk reduction among adults already taking maximally tolerated statins with triglyceride levels of 150 mg/dL or higher who have either established CVD or diabetes and two or more additional risk factors for CVD.
The approval is based largely on the REDUCE-IT trial’s finding of a 25% reduction in risk for major adverse cardiovascular events versus placebo. The FDA stated that the approval is the first for an agent with this specific indication.
Noting that it recognizes the need for additional medical treatments for CVD, the FDA says the new approval “will give patients with elevated triglycerides and other important risk factors, including heart disease, stroke, and diabetes, an adjunctive treatment option that can help decrease their risk of cardiovascular events.”
The drug was unanimously recommended for approval by the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee last month. But while the committee all agreed on its use in patients with established CVD, which made up 70% of the REDUCE-IT population, they were divided on whether the indication should be extended to the high-risk primary-prevention population, who made up just 30% of patients in the study.
Nonetheless, the FDA has gone for a broad indication based on the whole REDUCE-IT population.
In a conference call following the approval, Steven Ketchum, PhD, chief scientific officer at Amarin, pointed out that the primary-prevention population stipulated in the new approval differed very slightly from the REDUCE-IT enrollment criteria.
The trial specified that patients with diabetes should be older than 50 with one other cardiovascular risk factor, whereas the approved population is for diabetes and two cardiovascular risk factors. But as these two risk factors are not specified, they could include age, cigarette smoking, hypertension or use of an antihypertensive agent, low HDL cholesterol, high C-reactive protein, body mass index above 25 kg/m2, renal dysfunction, retinopathy, albuminuria, or an ankle branchial index below 0.9, Dr. Ketchum said.
“So while the label asks for two other risk factors, one of these could be age; so we believe the label is actually slightly broader than the REDUCE-IT inclusion criteria, and doctors have been left with significant leeway to decide which risk factors to consider on top of diabetes.”
Deepak Bhatt, MD, the lead investigator of REDUCE-IT, described the Vascepa approval as “the most significant event in the field of cardiovascular prevention since the introduction of statins nearly 3 decades ago.”
He commended the FDA on “a very evidence-based, prescriber-friendly, and most importantly, patient-friendly label,” which he said was in line with guidelines from multiple professional societies that have already incorporated the REDUCE-IT findings for secondary prevention and diabetic primary prevention.
Dr. Bhatt, who is a professor of medicine at Brigham and Women’s Hospital and Harvard Medical School, Boston, said the label essentially matches the REDUCE-IT population.
“The entry criteria for REDUCE-IT was fasting triglycerides greater than or equal to 150 mg/dL, with a 10% variance allowed (giving a minimum triglyceride value of 135 mg/dL). In actuality, we ended up with about 10% of the population with triglycerides between 100 and 150 mg/dL, and they had a similar degree of benefit as those with higher levels,” he reported.
“In the label, the 150 mg/dL does not specify fasting, and in fact many practices have moved away from fasting lipid measurements for the sake of patient comfort,” Dr. Bhatt added. “On average, nonfasting levels are about 50 mg/dL higher, so the label essentially mirrors those we studied, with the FDA applying good common sense and not being overly dogmatic about the exact wording of the trial inclusion criteria.”
No price change foreseen
Vascepa is already on the market for patients with very high triglyceride levels, and the company says it is not increasing the current price of about $300 a month, which is “relatively low, compared to other new breakthrough drugs.” However, it says it expects sales to grow from vastly increased volume based on the new indication.
Dr. Bhatt noted that REDUCE-IT cost-effectiveness data presented at the recent American Heart Association scientific sessions found the drug to be cost saving in the majority of cases. “That is something that is quite rare in cost-effectiveness research,” he said.
“Now, the key challenge is to identify and treat appropriate patients,” Dr. Bhatt noted. He says this task will largely fall on cardiologists, endocrinologists, and primary care physicians, though stroke neurologists, nephrologists, and vascular medicine specialists will also have patients for whom the data are relevant.
“I believe the drug will ultimately be widely prescribed, initially by subspecialists, but by primary care physicians also. It is overall very well tolerated, safe, and easy to use,” he said. “Much like statin prescription started in subspecialty practices but then became quite common in primary care, I envision the same happening with icosapent ethyl.”
Lipid expert Roger Blumenthal, MD, who was not involved in the REDUCE-IT trial, also welcomed the new approval for Vascepa.
“The indication is very appropriate; it is great to have another disease-modifying medication in our prevention toolkit,” Dr. Blumenthal, who is director of the Johns Hopkins Ciccarone Center for the Prevention of Cardiovascular Disease in Baltimore, said in an interview.
Some still unsure
But not everyone is in full agreement with the broad indication granted.
One expert who has reservations is James de Lemos, MD, professor of medicine at the University of Texas, Dallas, who sat on the FDA advisory committee that assessed the drug last month.
“I would have preferred a narrower label for now, limited to the secondary prevention indication, because I felt that REDUCE-IT did not include sufficient numbers of patients to justify the high-risk primary-prevention indication. We need an adequately powered, randomized, controlled trial to establish the risk/benefit and cost/benefit in primary prevention, and with this broad label, I worry there will be little incentive for the company to pursue this,” Dr. de Lemos commented in an interview.
“This is a slippery slope, and we should not allow broad indications that extend to primary prevention for drugs that were studied in mixed secondary- and primary-prevention patients, with the results driven by the secondary-prevention subset. These two subgroups are fundamentally different populations in whom the pathophysiology and the background treatments are very different,” he added.
However, Dr. de Lemos acknowledged that he would use Vascepa for some high-risk primary-prevention patients in his practice – those with diabetes, high triglycerides, and multiple risk factors. “I just wish we had more data coming so that I could be more certain of the benefit in this group,” he said.
Dr. Bhatt disclosed sitting on advisory boards for Cardax, Cereno Scientific, Medscape Cardiology, PhaseBio, and Regado Biosciences; conducting unfunded research in association with FlowCo, Fractyl, Merck, Novo Nordisk, PLx Pharma, and Takeda; and receiving research funding from Abbott, Afimmune, Amarin, Amgen, AstraZeneca, Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, Chiesi, CSL Behring, Eisai, Ethicon, Ferring Pharmaceuticals, and Forest Laboratories. Dr. De Lemos reported receiving grant and consulting income from Roche Diagnostics and Abbott Diagnostics; and consulting for Ortho Clinical Diagnostics. Dr. Blumenthal has disclosed no relevant financial relationships.
This article first appeared on Medscape.com.
The newly approved U.S. indication for icosapent ethyl (Vascepa; Amarin) is broadly in line with the entry criteria for the REDUCE-IT trial and includes a large high-risk primary-prevention population, as well as those with established cardiovascular disease (CVD). The drug, thus, could well be used by millions of patients in the United States alone.
The high-dose, purified eicosapentaenoic acid product was approved last week by the Food and Drug Administration for cardiovascular risk reduction among adults already taking maximally tolerated statins with triglyceride levels of 150 mg/dL or higher who have either established CVD or diabetes and two or more additional risk factors for CVD.
The approval is based largely on the REDUCE-IT trial’s finding of a 25% reduction in risk for major adverse cardiovascular events versus placebo. The FDA stated that the approval is the first for an agent with this specific indication.
Noting that it recognizes the need for additional medical treatments for CVD, the FDA says the new approval “will give patients with elevated triglycerides and other important risk factors, including heart disease, stroke, and diabetes, an adjunctive treatment option that can help decrease their risk of cardiovascular events.”
The drug was unanimously recommended for approval by the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee last month. But while the committee all agreed on its use in patients with established CVD, which made up 70% of the REDUCE-IT population, they were divided on whether the indication should be extended to the high-risk primary-prevention population, who made up just 30% of patients in the study.
Nonetheless, the FDA has gone for a broad indication based on the whole REDUCE-IT population.
In a conference call following the approval, Steven Ketchum, PhD, chief scientific officer at Amarin, pointed out that the primary-prevention population stipulated in the new approval differed very slightly from the REDUCE-IT enrollment criteria.
The trial specified that patients with diabetes should be older than 50 with one other cardiovascular risk factor, whereas the approved population is for diabetes and two cardiovascular risk factors. But as these two risk factors are not specified, they could include age, cigarette smoking, hypertension or use of an antihypertensive agent, low HDL cholesterol, high C-reactive protein, body mass index above 25 kg/m2, renal dysfunction, retinopathy, albuminuria, or an ankle branchial index below 0.9, Dr. Ketchum said.
“So while the label asks for two other risk factors, one of these could be age; so we believe the label is actually slightly broader than the REDUCE-IT inclusion criteria, and doctors have been left with significant leeway to decide which risk factors to consider on top of diabetes.”
Deepak Bhatt, MD, the lead investigator of REDUCE-IT, described the Vascepa approval as “the most significant event in the field of cardiovascular prevention since the introduction of statins nearly 3 decades ago.”
He commended the FDA on “a very evidence-based, prescriber-friendly, and most importantly, patient-friendly label,” which he said was in line with guidelines from multiple professional societies that have already incorporated the REDUCE-IT findings for secondary prevention and diabetic primary prevention.
Dr. Bhatt, who is a professor of medicine at Brigham and Women’s Hospital and Harvard Medical School, Boston, said the label essentially matches the REDUCE-IT population.
“The entry criteria for REDUCE-IT was fasting triglycerides greater than or equal to 150 mg/dL, with a 10% variance allowed (giving a minimum triglyceride value of 135 mg/dL). In actuality, we ended up with about 10% of the population with triglycerides between 100 and 150 mg/dL, and they had a similar degree of benefit as those with higher levels,” he reported.
“In the label, the 150 mg/dL does not specify fasting, and in fact many practices have moved away from fasting lipid measurements for the sake of patient comfort,” Dr. Bhatt added. “On average, nonfasting levels are about 50 mg/dL higher, so the label essentially mirrors those we studied, with the FDA applying good common sense and not being overly dogmatic about the exact wording of the trial inclusion criteria.”
No price change foreseen
Vascepa is already on the market for patients with very high triglyceride levels, and the company says it is not increasing the current price of about $300 a month, which is “relatively low, compared to other new breakthrough drugs.” However, it says it expects sales to grow from vastly increased volume based on the new indication.
Dr. Bhatt noted that REDUCE-IT cost-effectiveness data presented at the recent American Heart Association scientific sessions found the drug to be cost saving in the majority of cases. “That is something that is quite rare in cost-effectiveness research,” he said.
“Now, the key challenge is to identify and treat appropriate patients,” Dr. Bhatt noted. He says this task will largely fall on cardiologists, endocrinologists, and primary care physicians, though stroke neurologists, nephrologists, and vascular medicine specialists will also have patients for whom the data are relevant.
“I believe the drug will ultimately be widely prescribed, initially by subspecialists, but by primary care physicians also. It is overall very well tolerated, safe, and easy to use,” he said. “Much like statin prescription started in subspecialty practices but then became quite common in primary care, I envision the same happening with icosapent ethyl.”
Lipid expert Roger Blumenthal, MD, who was not involved in the REDUCE-IT trial, also welcomed the new approval for Vascepa.
“The indication is very appropriate; it is great to have another disease-modifying medication in our prevention toolkit,” Dr. Blumenthal, who is director of the Johns Hopkins Ciccarone Center for the Prevention of Cardiovascular Disease in Baltimore, said in an interview.
Some still unsure
But not everyone is in full agreement with the broad indication granted.
One expert who has reservations is James de Lemos, MD, professor of medicine at the University of Texas, Dallas, who sat on the FDA advisory committee that assessed the drug last month.
“I would have preferred a narrower label for now, limited to the secondary prevention indication, because I felt that REDUCE-IT did not include sufficient numbers of patients to justify the high-risk primary-prevention indication. We need an adequately powered, randomized, controlled trial to establish the risk/benefit and cost/benefit in primary prevention, and with this broad label, I worry there will be little incentive for the company to pursue this,” Dr. de Lemos commented in an interview.
“This is a slippery slope, and we should not allow broad indications that extend to primary prevention for drugs that were studied in mixed secondary- and primary-prevention patients, with the results driven by the secondary-prevention subset. These two subgroups are fundamentally different populations in whom the pathophysiology and the background treatments are very different,” he added.
However, Dr. de Lemos acknowledged that he would use Vascepa for some high-risk primary-prevention patients in his practice – those with diabetes, high triglycerides, and multiple risk factors. “I just wish we had more data coming so that I could be more certain of the benefit in this group,” he said.
Dr. Bhatt disclosed sitting on advisory boards for Cardax, Cereno Scientific, Medscape Cardiology, PhaseBio, and Regado Biosciences; conducting unfunded research in association with FlowCo, Fractyl, Merck, Novo Nordisk, PLx Pharma, and Takeda; and receiving research funding from Abbott, Afimmune, Amarin, Amgen, AstraZeneca, Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, Chiesi, CSL Behring, Eisai, Ethicon, Ferring Pharmaceuticals, and Forest Laboratories. Dr. De Lemos reported receiving grant and consulting income from Roche Diagnostics and Abbott Diagnostics; and consulting for Ortho Clinical Diagnostics. Dr. Blumenthal has disclosed no relevant financial relationships.
This article first appeared on Medscape.com.
The newly approved U.S. indication for icosapent ethyl (Vascepa; Amarin) is broadly in line with the entry criteria for the REDUCE-IT trial and includes a large high-risk primary-prevention population, as well as those with established cardiovascular disease (CVD). The drug, thus, could well be used by millions of patients in the United States alone.
The high-dose, purified eicosapentaenoic acid product was approved last week by the Food and Drug Administration for cardiovascular risk reduction among adults already taking maximally tolerated statins with triglyceride levels of 150 mg/dL or higher who have either established CVD or diabetes and two or more additional risk factors for CVD.
The approval is based largely on the REDUCE-IT trial’s finding of a 25% reduction in risk for major adverse cardiovascular events versus placebo. The FDA stated that the approval is the first for an agent with this specific indication.
Noting that it recognizes the need for additional medical treatments for CVD, the FDA says the new approval “will give patients with elevated triglycerides and other important risk factors, including heart disease, stroke, and diabetes, an adjunctive treatment option that can help decrease their risk of cardiovascular events.”
The drug was unanimously recommended for approval by the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee last month. But while the committee all agreed on its use in patients with established CVD, which made up 70% of the REDUCE-IT population, they were divided on whether the indication should be extended to the high-risk primary-prevention population, who made up just 30% of patients in the study.
Nonetheless, the FDA has gone for a broad indication based on the whole REDUCE-IT population.
In a conference call following the approval, Steven Ketchum, PhD, chief scientific officer at Amarin, pointed out that the primary-prevention population stipulated in the new approval differed very slightly from the REDUCE-IT enrollment criteria.
The trial specified that patients with diabetes should be older than 50 with one other cardiovascular risk factor, whereas the approved population is for diabetes and two cardiovascular risk factors. But as these two risk factors are not specified, they could include age, cigarette smoking, hypertension or use of an antihypertensive agent, low HDL cholesterol, high C-reactive protein, body mass index above 25 kg/m2, renal dysfunction, retinopathy, albuminuria, or an ankle branchial index below 0.9, Dr. Ketchum said.
“So while the label asks for two other risk factors, one of these could be age; so we believe the label is actually slightly broader than the REDUCE-IT inclusion criteria, and doctors have been left with significant leeway to decide which risk factors to consider on top of diabetes.”
Deepak Bhatt, MD, the lead investigator of REDUCE-IT, described the Vascepa approval as “the most significant event in the field of cardiovascular prevention since the introduction of statins nearly 3 decades ago.”
He commended the FDA on “a very evidence-based, prescriber-friendly, and most importantly, patient-friendly label,” which he said was in line with guidelines from multiple professional societies that have already incorporated the REDUCE-IT findings for secondary prevention and diabetic primary prevention.
Dr. Bhatt, who is a professor of medicine at Brigham and Women’s Hospital and Harvard Medical School, Boston, said the label essentially matches the REDUCE-IT population.
“The entry criteria for REDUCE-IT was fasting triglycerides greater than or equal to 150 mg/dL, with a 10% variance allowed (giving a minimum triglyceride value of 135 mg/dL). In actuality, we ended up with about 10% of the population with triglycerides between 100 and 150 mg/dL, and they had a similar degree of benefit as those with higher levels,” he reported.
“In the label, the 150 mg/dL does not specify fasting, and in fact many practices have moved away from fasting lipid measurements for the sake of patient comfort,” Dr. Bhatt added. “On average, nonfasting levels are about 50 mg/dL higher, so the label essentially mirrors those we studied, with the FDA applying good common sense and not being overly dogmatic about the exact wording of the trial inclusion criteria.”
No price change foreseen
Vascepa is already on the market for patients with very high triglyceride levels, and the company says it is not increasing the current price of about $300 a month, which is “relatively low, compared to other new breakthrough drugs.” However, it says it expects sales to grow from vastly increased volume based on the new indication.
Dr. Bhatt noted that REDUCE-IT cost-effectiveness data presented at the recent American Heart Association scientific sessions found the drug to be cost saving in the majority of cases. “That is something that is quite rare in cost-effectiveness research,” he said.
“Now, the key challenge is to identify and treat appropriate patients,” Dr. Bhatt noted. He says this task will largely fall on cardiologists, endocrinologists, and primary care physicians, though stroke neurologists, nephrologists, and vascular medicine specialists will also have patients for whom the data are relevant.
“I believe the drug will ultimately be widely prescribed, initially by subspecialists, but by primary care physicians also. It is overall very well tolerated, safe, and easy to use,” he said. “Much like statin prescription started in subspecialty practices but then became quite common in primary care, I envision the same happening with icosapent ethyl.”
Lipid expert Roger Blumenthal, MD, who was not involved in the REDUCE-IT trial, also welcomed the new approval for Vascepa.
“The indication is very appropriate; it is great to have another disease-modifying medication in our prevention toolkit,” Dr. Blumenthal, who is director of the Johns Hopkins Ciccarone Center for the Prevention of Cardiovascular Disease in Baltimore, said in an interview.
Some still unsure
But not everyone is in full agreement with the broad indication granted.
One expert who has reservations is James de Lemos, MD, professor of medicine at the University of Texas, Dallas, who sat on the FDA advisory committee that assessed the drug last month.
“I would have preferred a narrower label for now, limited to the secondary prevention indication, because I felt that REDUCE-IT did not include sufficient numbers of patients to justify the high-risk primary-prevention indication. We need an adequately powered, randomized, controlled trial to establish the risk/benefit and cost/benefit in primary prevention, and with this broad label, I worry there will be little incentive for the company to pursue this,” Dr. de Lemos commented in an interview.
“This is a slippery slope, and we should not allow broad indications that extend to primary prevention for drugs that were studied in mixed secondary- and primary-prevention patients, with the results driven by the secondary-prevention subset. These two subgroups are fundamentally different populations in whom the pathophysiology and the background treatments are very different,” he added.
However, Dr. de Lemos acknowledged that he would use Vascepa for some high-risk primary-prevention patients in his practice – those with diabetes, high triglycerides, and multiple risk factors. “I just wish we had more data coming so that I could be more certain of the benefit in this group,” he said.
Dr. Bhatt disclosed sitting on advisory boards for Cardax, Cereno Scientific, Medscape Cardiology, PhaseBio, and Regado Biosciences; conducting unfunded research in association with FlowCo, Fractyl, Merck, Novo Nordisk, PLx Pharma, and Takeda; and receiving research funding from Abbott, Afimmune, Amarin, Amgen, AstraZeneca, Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, Chiesi, CSL Behring, Eisai, Ethicon, Ferring Pharmaceuticals, and Forest Laboratories. Dr. De Lemos reported receiving grant and consulting income from Roche Diagnostics and Abbott Diagnostics; and consulting for Ortho Clinical Diagnostics. Dr. Blumenthal has disclosed no relevant financial relationships.
This article first appeared on Medscape.com.
FDA approves first treatment for advanced epithelioid sarcoma
The Food and Drug Administration has granted accelerated approval to tazemetostat (Tazverik) for the treatment of adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection.
Approval was based on overall response rate in a trial enrolling 62 patients with metastatic or locally advanced epithelioid sarcoma. The overall response rate was 15%, with 1.6% of patients having a complete response and 13% having a partial response. Of the nine patients that had a response, six (67%) had a response lasting 6 months or longer, the FDA said in a press statement.
The most common side effects for patients taking tazemetostat were pain, fatigue, nausea, decreased appetite, vomiting, and constipation. Patients treated with tazemetostat are at increased risk of developing secondary malignancies, including T-cell lymphoblastic lymphoma, myelodysplastic syndrome, and acute myeloid leukemia.
“Epithelioid sarcoma accounts for less than 1% of all soft-tissue sarcomas,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the Center for Drug Evaluation and Research. “Until today, there were no treatment options specifically for patients with epithelioid sarcoma. The approval of Tazverik provides a treatment option that specifically targets this disease.”
Tazemetostat must be dispensed with a patient medication guide that describes important information about the drug’s uses and risks, the FDA said.
The Food and Drug Administration has granted accelerated approval to tazemetostat (Tazverik) for the treatment of adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection.
Approval was based on overall response rate in a trial enrolling 62 patients with metastatic or locally advanced epithelioid sarcoma. The overall response rate was 15%, with 1.6% of patients having a complete response and 13% having a partial response. Of the nine patients that had a response, six (67%) had a response lasting 6 months or longer, the FDA said in a press statement.
The most common side effects for patients taking tazemetostat were pain, fatigue, nausea, decreased appetite, vomiting, and constipation. Patients treated with tazemetostat are at increased risk of developing secondary malignancies, including T-cell lymphoblastic lymphoma, myelodysplastic syndrome, and acute myeloid leukemia.
“Epithelioid sarcoma accounts for less than 1% of all soft-tissue sarcomas,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the Center for Drug Evaluation and Research. “Until today, there were no treatment options specifically for patients with epithelioid sarcoma. The approval of Tazverik provides a treatment option that specifically targets this disease.”
Tazemetostat must be dispensed with a patient medication guide that describes important information about the drug’s uses and risks, the FDA said.
The Food and Drug Administration has granted accelerated approval to tazemetostat (Tazverik) for the treatment of adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection.
Approval was based on overall response rate in a trial enrolling 62 patients with metastatic or locally advanced epithelioid sarcoma. The overall response rate was 15%, with 1.6% of patients having a complete response and 13% having a partial response. Of the nine patients that had a response, six (67%) had a response lasting 6 months or longer, the FDA said in a press statement.
The most common side effects for patients taking tazemetostat were pain, fatigue, nausea, decreased appetite, vomiting, and constipation. Patients treated with tazemetostat are at increased risk of developing secondary malignancies, including T-cell lymphoblastic lymphoma, myelodysplastic syndrome, and acute myeloid leukemia.
“Epithelioid sarcoma accounts for less than 1% of all soft-tissue sarcomas,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the Center for Drug Evaluation and Research. “Until today, there were no treatment options specifically for patients with epithelioid sarcoma. The approval of Tazverik provides a treatment option that specifically targets this disease.”
Tazemetostat must be dispensed with a patient medication guide that describes important information about the drug’s uses and risks, the FDA said.
Teprotumumab gets FDA go-ahead for thyroid eye disease
according to a press release.
Thyroid eye disease is a rare, progressive, autoimmune condition that causes the eyes to bulge (proptosis) and can lead to blindness. Until now, treatment has focused on managing its symptoms – which can include eye pain, double vision, or sensitivity to light – with steroids, and in some cases, multiple invasive surgeries.
The human monoclonal antibody and a targeted inhibitor of the insulinlike growth factor-1 receptor is administered to patients once every 3 weeks, for a total of eight infusions, according to a statement from Horizon Therapeutics, which manufactures the drug.
The approval was based on the findings from two similarly designed, parallel-group studies (Studies 1 and 2) involving 170 patients with thyroid eye disease who were randomized to receive either teprotumumab or placebo. Of those receiving the study drug, 71% in Study 1 and 83% in Study 2 had a reduction of more than 2 mm in eye protrusion, compared with 20% and 10%, respectively, among the placebo participants.
The most common adverse reactions in patients receiving teprotumumab were muscle spasm, nausea, alopecia, diarrhea, fatigue, and hyperglycemia. The treatment is contraindicated for pregnancy.
according to a press release.
Thyroid eye disease is a rare, progressive, autoimmune condition that causes the eyes to bulge (proptosis) and can lead to blindness. Until now, treatment has focused on managing its symptoms – which can include eye pain, double vision, or sensitivity to light – with steroids, and in some cases, multiple invasive surgeries.
The human monoclonal antibody and a targeted inhibitor of the insulinlike growth factor-1 receptor is administered to patients once every 3 weeks, for a total of eight infusions, according to a statement from Horizon Therapeutics, which manufactures the drug.
The approval was based on the findings from two similarly designed, parallel-group studies (Studies 1 and 2) involving 170 patients with thyroid eye disease who were randomized to receive either teprotumumab or placebo. Of those receiving the study drug, 71% in Study 1 and 83% in Study 2 had a reduction of more than 2 mm in eye protrusion, compared with 20% and 10%, respectively, among the placebo participants.
The most common adverse reactions in patients receiving teprotumumab were muscle spasm, nausea, alopecia, diarrhea, fatigue, and hyperglycemia. The treatment is contraindicated for pregnancy.
according to a press release.
Thyroid eye disease is a rare, progressive, autoimmune condition that causes the eyes to bulge (proptosis) and can lead to blindness. Until now, treatment has focused on managing its symptoms – which can include eye pain, double vision, or sensitivity to light – with steroids, and in some cases, multiple invasive surgeries.
The human monoclonal antibody and a targeted inhibitor of the insulinlike growth factor-1 receptor is administered to patients once every 3 weeks, for a total of eight infusions, according to a statement from Horizon Therapeutics, which manufactures the drug.
The approval was based on the findings from two similarly designed, parallel-group studies (Studies 1 and 2) involving 170 patients with thyroid eye disease who were randomized to receive either teprotumumab or placebo. Of those receiving the study drug, 71% in Study 1 and 83% in Study 2 had a reduction of more than 2 mm in eye protrusion, compared with 20% and 10%, respectively, among the placebo participants.
The most common adverse reactions in patients receiving teprotumumab were muscle spasm, nausea, alopecia, diarrhea, fatigue, and hyperglycemia. The treatment is contraindicated for pregnancy.
FROM THE FDA
FDA approves CV disease benefit for once-weekly semaglutide
(Ozempic, Novo Nordisk) in the treatment of type 2 diabetes and has added new trial data information to the label of the oral version (Rybelsus, Novo Nordisk) pertaining to CV safety.
The agency expanded the once-weekly injectable semaglutide’s label to include an indication for reducing the risk for major adverse cardiovascular events (MACE), including CV death, nonfatal myocardial infarction (MI), or nonfatal stroke, in adults with type 2 diabetes who have established CV disease.
Initially approved for the treatment of type 2 diabetes in adults in December 2017, once-weekly subcutaneously injectable semaglutide comes in 0.5-mg and 1.0-mg doses in a dedicated prefilled pen device.
The approval for the new indication was based on data from the 2-year randomized Trial to Evaluate Cardiovascular and Other Long-term Outcomes With Semaglutide in Subjects With Type 2 Diabetes (SUSTAIN 6), in which injectable semaglutide or placebo was added to standard of care in 3,297 adults with type 2 diabetes and established CV disease.
As reported by Medscape Medical News in September 2016, patients who underwent treatment with one of the two doses of semaglutide had a significant 26% lower risk for the primary composite outcome of first occurrence of CV death, nonfatal MI, or nonfatal stroke, compared with those who received placebo.
The reduced CV risk was driven primarily by significant reductions in nonfatal stroke and nonfatal MI (39% and 26%, respectively). There was no difference in CV death between drug and placebo groups. Gastrointestinal adverse events were more frequent with semaglutide than placebo, with the majority occurring during the first 30 weeks.
The oral semaglutide formulation, taken daily in 7-mg or 14-mg doses, was approved for the treatment of type 2 diabetes in September 2019, the first orally available GLP-1 agonist.
Now, the FDA has updated the prescribing information in the clinical studies section (section 14) to include results from the randomized, placebo-controlled, 3,183-subject Trial Investigating the Cardiovascular Safety of Oral Semaglutide in Subjects With Type 2 Diabetes (PIONEER 6), reported in June 2019, which found a nonsignificant 21% reduction in three-component MACE with oral semaglutide. The addition to the label for Rybelsus is with regard to CV safety, not benefit.
SOUL: Large ongoing CV outcomes trial for oral semaglutide
In June 2019, Novo Nordisk initiated a larger CV outcomes trial of oral semaglutide, A Heart Disease Study of Semaglutide in Patients With Type 2 Diabetes (SOUL). The trial, which includes 9,642 adults with type 2 diabetes and established CV disease, is further investigating the drug’s effects on the incidence of MACE. The estimated completion date is July 2024.
Semaglutide joins a growing list of drugs approved for treating type 2 diabetes that have been granted additional label indications for benefits beyond the lowering of glucose.
These include Novo Nordisk’s other injectable GLP-1 agonist, liraglutide (Victoza), additionally approved for reducing CV events in high-risk patients with type 2 diabetes, and the sodium-glucose transport 2 inhibitors empagliflozin (Jardiance, Boehringer Ingelheim/Lilly), for reducing CV death; canagliflozin (Invokana, Janssen), for reducing major adverse CV events,; kidney disease, CV death, and heart failure hospitalization; and dapagliflozin (Farxiga, AstraZeneca), for reducing heart failure hospitalization.
Recently, in an exclusive Medscape Medical News article, some experts questioned the design of these CV outcomes trials, suggesting that imbalances in glycemic control, blood pressure, and diuretic use between treatment and placebo arms could have biased the CV and renal outcomes of the trials in favor of the study drugs
A version of this story originally appeared on Medscape.com.
(Ozempic, Novo Nordisk) in the treatment of type 2 diabetes and has added new trial data information to the label of the oral version (Rybelsus, Novo Nordisk) pertaining to CV safety.
The agency expanded the once-weekly injectable semaglutide’s label to include an indication for reducing the risk for major adverse cardiovascular events (MACE), including CV death, nonfatal myocardial infarction (MI), or nonfatal stroke, in adults with type 2 diabetes who have established CV disease.
Initially approved for the treatment of type 2 diabetes in adults in December 2017, once-weekly subcutaneously injectable semaglutide comes in 0.5-mg and 1.0-mg doses in a dedicated prefilled pen device.
The approval for the new indication was based on data from the 2-year randomized Trial to Evaluate Cardiovascular and Other Long-term Outcomes With Semaglutide in Subjects With Type 2 Diabetes (SUSTAIN 6), in which injectable semaglutide or placebo was added to standard of care in 3,297 adults with type 2 diabetes and established CV disease.
As reported by Medscape Medical News in September 2016, patients who underwent treatment with one of the two doses of semaglutide had a significant 26% lower risk for the primary composite outcome of first occurrence of CV death, nonfatal MI, or nonfatal stroke, compared with those who received placebo.
The reduced CV risk was driven primarily by significant reductions in nonfatal stroke and nonfatal MI (39% and 26%, respectively). There was no difference in CV death between drug and placebo groups. Gastrointestinal adverse events were more frequent with semaglutide than placebo, with the majority occurring during the first 30 weeks.
The oral semaglutide formulation, taken daily in 7-mg or 14-mg doses, was approved for the treatment of type 2 diabetes in September 2019, the first orally available GLP-1 agonist.
Now, the FDA has updated the prescribing information in the clinical studies section (section 14) to include results from the randomized, placebo-controlled, 3,183-subject Trial Investigating the Cardiovascular Safety of Oral Semaglutide in Subjects With Type 2 Diabetes (PIONEER 6), reported in June 2019, which found a nonsignificant 21% reduction in three-component MACE with oral semaglutide. The addition to the label for Rybelsus is with regard to CV safety, not benefit.
SOUL: Large ongoing CV outcomes trial for oral semaglutide
In June 2019, Novo Nordisk initiated a larger CV outcomes trial of oral semaglutide, A Heart Disease Study of Semaglutide in Patients With Type 2 Diabetes (SOUL). The trial, which includes 9,642 adults with type 2 diabetes and established CV disease, is further investigating the drug’s effects on the incidence of MACE. The estimated completion date is July 2024.
Semaglutide joins a growing list of drugs approved for treating type 2 diabetes that have been granted additional label indications for benefits beyond the lowering of glucose.
These include Novo Nordisk’s other injectable GLP-1 agonist, liraglutide (Victoza), additionally approved for reducing CV events in high-risk patients with type 2 diabetes, and the sodium-glucose transport 2 inhibitors empagliflozin (Jardiance, Boehringer Ingelheim/Lilly), for reducing CV death; canagliflozin (Invokana, Janssen), for reducing major adverse CV events,; kidney disease, CV death, and heart failure hospitalization; and dapagliflozin (Farxiga, AstraZeneca), for reducing heart failure hospitalization.
Recently, in an exclusive Medscape Medical News article, some experts questioned the design of these CV outcomes trials, suggesting that imbalances in glycemic control, blood pressure, and diuretic use between treatment and placebo arms could have biased the CV and renal outcomes of the trials in favor of the study drugs
A version of this story originally appeared on Medscape.com.
(Ozempic, Novo Nordisk) in the treatment of type 2 diabetes and has added new trial data information to the label of the oral version (Rybelsus, Novo Nordisk) pertaining to CV safety.
The agency expanded the once-weekly injectable semaglutide’s label to include an indication for reducing the risk for major adverse cardiovascular events (MACE), including CV death, nonfatal myocardial infarction (MI), or nonfatal stroke, in adults with type 2 diabetes who have established CV disease.
Initially approved for the treatment of type 2 diabetes in adults in December 2017, once-weekly subcutaneously injectable semaglutide comes in 0.5-mg and 1.0-mg doses in a dedicated prefilled pen device.
The approval for the new indication was based on data from the 2-year randomized Trial to Evaluate Cardiovascular and Other Long-term Outcomes With Semaglutide in Subjects With Type 2 Diabetes (SUSTAIN 6), in which injectable semaglutide or placebo was added to standard of care in 3,297 adults with type 2 diabetes and established CV disease.
As reported by Medscape Medical News in September 2016, patients who underwent treatment with one of the two doses of semaglutide had a significant 26% lower risk for the primary composite outcome of first occurrence of CV death, nonfatal MI, or nonfatal stroke, compared with those who received placebo.
The reduced CV risk was driven primarily by significant reductions in nonfatal stroke and nonfatal MI (39% and 26%, respectively). There was no difference in CV death between drug and placebo groups. Gastrointestinal adverse events were more frequent with semaglutide than placebo, with the majority occurring during the first 30 weeks.
The oral semaglutide formulation, taken daily in 7-mg or 14-mg doses, was approved for the treatment of type 2 diabetes in September 2019, the first orally available GLP-1 agonist.
Now, the FDA has updated the prescribing information in the clinical studies section (section 14) to include results from the randomized, placebo-controlled, 3,183-subject Trial Investigating the Cardiovascular Safety of Oral Semaglutide in Subjects With Type 2 Diabetes (PIONEER 6), reported in June 2019, which found a nonsignificant 21% reduction in three-component MACE with oral semaglutide. The addition to the label for Rybelsus is with regard to CV safety, not benefit.
SOUL: Large ongoing CV outcomes trial for oral semaglutide
In June 2019, Novo Nordisk initiated a larger CV outcomes trial of oral semaglutide, A Heart Disease Study of Semaglutide in Patients With Type 2 Diabetes (SOUL). The trial, which includes 9,642 adults with type 2 diabetes and established CV disease, is further investigating the drug’s effects on the incidence of MACE. The estimated completion date is July 2024.
Semaglutide joins a growing list of drugs approved for treating type 2 diabetes that have been granted additional label indications for benefits beyond the lowering of glucose.
These include Novo Nordisk’s other injectable GLP-1 agonist, liraglutide (Victoza), additionally approved for reducing CV events in high-risk patients with type 2 diabetes, and the sodium-glucose transport 2 inhibitors empagliflozin (Jardiance, Boehringer Ingelheim/Lilly), for reducing CV death; canagliflozin (Invokana, Janssen), for reducing major adverse CV events,; kidney disease, CV death, and heart failure hospitalization; and dapagliflozin (Farxiga, AstraZeneca), for reducing heart failure hospitalization.
Recently, in an exclusive Medscape Medical News article, some experts questioned the design of these CV outcomes trials, suggesting that imbalances in glycemic control, blood pressure, and diuretic use between treatment and placebo arms could have biased the CV and renal outcomes of the trials in favor of the study drugs
A version of this story originally appeared on Medscape.com.
FDA warns of possible cancer risk with lorcaserin
after results from a clinical trial assessing the drug’s safety showed a possible increased risk of cancer.
“At this time, the cause of the cancer is uncertain, and we cannot conclude that lorcaserin contributes to the cancer risk. However, we wanted to make the public aware of this potential risk,” the agency said in a press release.
The agency advised that health care providers consider whether the benefits of taking lorcaserin outweighed the potential cancer risk, and that patients currently taking the medication should talk to their providers about the risks.
“We are continuing to evaluate the clinical trial results and will communicate our final conclusions and recommendations when we have completed our review,” the FDA noted in the statement.
Lorcaserin, a serotonin 2C receptor agonist, was approved by the FDA in 2012 at a dosage of 20 mg once daily for use with a reduced-calorie diet and increased physical activity as a means to improve weight loss in adults who are obese or overweight and have at least one weight-related medical problem, such as such as hypertension, type 2 diabetes, or dyslipidemia. In July 2016, the agency approved a New Drug Application for an extended-release, once-daily formulation.
Headache, dizziness, fatigue, nausea, dry mouth, and constipation are the more common adverse effects in patients without diabetes, whereas hypoglycemia, headache, back pain, cough, and fatigue are more common in patients with diabetes. The treatment is contraindicated for pregnancy.
Lorcaserin is distributed by Eisai.*
*Correction, 1/15/2020: An earlier version of this story misstated the manufacturer of lorcaserin.
after results from a clinical trial assessing the drug’s safety showed a possible increased risk of cancer.
“At this time, the cause of the cancer is uncertain, and we cannot conclude that lorcaserin contributes to the cancer risk. However, we wanted to make the public aware of this potential risk,” the agency said in a press release.
The agency advised that health care providers consider whether the benefits of taking lorcaserin outweighed the potential cancer risk, and that patients currently taking the medication should talk to their providers about the risks.
“We are continuing to evaluate the clinical trial results and will communicate our final conclusions and recommendations when we have completed our review,” the FDA noted in the statement.
Lorcaserin, a serotonin 2C receptor agonist, was approved by the FDA in 2012 at a dosage of 20 mg once daily for use with a reduced-calorie diet and increased physical activity as a means to improve weight loss in adults who are obese or overweight and have at least one weight-related medical problem, such as such as hypertension, type 2 diabetes, or dyslipidemia. In July 2016, the agency approved a New Drug Application for an extended-release, once-daily formulation.
Headache, dizziness, fatigue, nausea, dry mouth, and constipation are the more common adverse effects in patients without diabetes, whereas hypoglycemia, headache, back pain, cough, and fatigue are more common in patients with diabetes. The treatment is contraindicated for pregnancy.
Lorcaserin is distributed by Eisai.*
*Correction, 1/15/2020: An earlier version of this story misstated the manufacturer of lorcaserin.
after results from a clinical trial assessing the drug’s safety showed a possible increased risk of cancer.
“At this time, the cause of the cancer is uncertain, and we cannot conclude that lorcaserin contributes to the cancer risk. However, we wanted to make the public aware of this potential risk,” the agency said in a press release.
The agency advised that health care providers consider whether the benefits of taking lorcaserin outweighed the potential cancer risk, and that patients currently taking the medication should talk to their providers about the risks.
“We are continuing to evaluate the clinical trial results and will communicate our final conclusions and recommendations when we have completed our review,” the FDA noted in the statement.
Lorcaserin, a serotonin 2C receptor agonist, was approved by the FDA in 2012 at a dosage of 20 mg once daily for use with a reduced-calorie diet and increased physical activity as a means to improve weight loss in adults who are obese or overweight and have at least one weight-related medical problem, such as such as hypertension, type 2 diabetes, or dyslipidemia. In July 2016, the agency approved a New Drug Application for an extended-release, once-daily formulation.
Headache, dizziness, fatigue, nausea, dry mouth, and constipation are the more common adverse effects in patients without diabetes, whereas hypoglycemia, headache, back pain, cough, and fatigue are more common in patients with diabetes. The treatment is contraindicated for pregnancy.
Lorcaserin is distributed by Eisai.*
*Correction, 1/15/2020: An earlier version of this story misstated the manufacturer of lorcaserin.
FDA approves avapritinib for adults with GIST with PDGFRA mutation
The Food and Drug Administration has approved avapritinib (Ayvakit) for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumors (GIST) with a platelet-derived growth factor receptor–alpha (PDGFRA) exon 18 mutation.
Approval was based on results from a clinical trial of 43 patients with PDGFRA exon 18 mutations, including 38 patients with a PDGFRA D842V mutation, who received 300 mg avapritinib once daily, the FDA said in a statement.
The overall response rate was 84% (7% with complete response, 77% with partial response); the response rate in patients with a D842V mutation was 89% (8% with complete response, 82% with partial response). Median response duration was not reached, but 61% of patients had a response lasting longer than 6 months.
The most common adverse events associated with avapritinib include edema, nausea, fatigue/asthenia, cognitive impairment, vomiting, decreased appetite, diarrhea, hair color changes, increased lacrimation, abdominal pain, constipation, rash, and dizziness. The drug also can cause intracranial hemorrhage and have effects on the central nervous system.
“GIST harboring a PDGFRA exon 18 mutation do not respond to standard therapies for GIST. However, today’s approval provides patients with the first drug specifically approved for GIST harboring this mutation,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the Center for Drug Evaluation and Research, said in the statement.
The Food and Drug Administration has approved avapritinib (Ayvakit) for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumors (GIST) with a platelet-derived growth factor receptor–alpha (PDGFRA) exon 18 mutation.
Approval was based on results from a clinical trial of 43 patients with PDGFRA exon 18 mutations, including 38 patients with a PDGFRA D842V mutation, who received 300 mg avapritinib once daily, the FDA said in a statement.
The overall response rate was 84% (7% with complete response, 77% with partial response); the response rate in patients with a D842V mutation was 89% (8% with complete response, 82% with partial response). Median response duration was not reached, but 61% of patients had a response lasting longer than 6 months.
The most common adverse events associated with avapritinib include edema, nausea, fatigue/asthenia, cognitive impairment, vomiting, decreased appetite, diarrhea, hair color changes, increased lacrimation, abdominal pain, constipation, rash, and dizziness. The drug also can cause intracranial hemorrhage and have effects on the central nervous system.
“GIST harboring a PDGFRA exon 18 mutation do not respond to standard therapies for GIST. However, today’s approval provides patients with the first drug specifically approved for GIST harboring this mutation,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the Center for Drug Evaluation and Research, said in the statement.
The Food and Drug Administration has approved avapritinib (Ayvakit) for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumors (GIST) with a platelet-derived growth factor receptor–alpha (PDGFRA) exon 18 mutation.
Approval was based on results from a clinical trial of 43 patients with PDGFRA exon 18 mutations, including 38 patients with a PDGFRA D842V mutation, who received 300 mg avapritinib once daily, the FDA said in a statement.
The overall response rate was 84% (7% with complete response, 77% with partial response); the response rate in patients with a D842V mutation was 89% (8% with complete response, 82% with partial response). Median response duration was not reached, but 61% of patients had a response lasting longer than 6 months.
The most common adverse events associated with avapritinib include edema, nausea, fatigue/asthenia, cognitive impairment, vomiting, decreased appetite, diarrhea, hair color changes, increased lacrimation, abdominal pain, constipation, rash, and dizziness. The drug also can cause intracranial hemorrhage and have effects on the central nervous system.
“GIST harboring a PDGFRA exon 18 mutation do not respond to standard therapies for GIST. However, today’s approval provides patients with the first drug specifically approved for GIST harboring this mutation,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the Center for Drug Evaluation and Research, said in the statement.
FDA Awards Grant To Study Temozolomide in Gist
A phase 2 study of temozolomide in gastrointestinal stromal tumors (GIST) received one of the 12 grants awarded in October by the US Food and Drug Administration (FDA) to enhance the development of medical products for patients with rare diseases. Jason Sicklick, MD, and the University of California San Diego in La Jolla will receive $1.5 million over 3 years to conduct the phase 2 study (NCT03556384). The objective of the study is to determine the efficacy at 6 months of temozolomide therapy in patients with the SDH-mutant/deficient subtype. Temozolomide is approved by the FDA to treat newly diagnosed glioblastoma multiforme and refractory anaplastic astrocytomas. It is not approved for the treatment of SDH-mutant/deficient GIST.
The FDA awarded the grants through the Orphan Products Clinical Trials Grants Program. Other orphan diseases receiving grants included glomerulopathy, gliomas, Fanconi anemia, sickle cell respiratory complications, Duchenne muscular dystrophy, HPV associated respiratory papillomatosis, refractory viral infections and T-cell immunodeficiency, oral cancer, retinoblastoma, cerebellar brain tumors, and acute myeloid leukemia.
A phase 2 study of temozolomide in gastrointestinal stromal tumors (GIST) received one of the 12 grants awarded in October by the US Food and Drug Administration (FDA) to enhance the development of medical products for patients with rare diseases. Jason Sicklick, MD, and the University of California San Diego in La Jolla will receive $1.5 million over 3 years to conduct the phase 2 study (NCT03556384). The objective of the study is to determine the efficacy at 6 months of temozolomide therapy in patients with the SDH-mutant/deficient subtype. Temozolomide is approved by the FDA to treat newly diagnosed glioblastoma multiforme and refractory anaplastic astrocytomas. It is not approved for the treatment of SDH-mutant/deficient GIST.
The FDA awarded the grants through the Orphan Products Clinical Trials Grants Program. Other orphan diseases receiving grants included glomerulopathy, gliomas, Fanconi anemia, sickle cell respiratory complications, Duchenne muscular dystrophy, HPV associated respiratory papillomatosis, refractory viral infections and T-cell immunodeficiency, oral cancer, retinoblastoma, cerebellar brain tumors, and acute myeloid leukemia.
A phase 2 study of temozolomide in gastrointestinal stromal tumors (GIST) received one of the 12 grants awarded in October by the US Food and Drug Administration (FDA) to enhance the development of medical products for patients with rare diseases. Jason Sicklick, MD, and the University of California San Diego in La Jolla will receive $1.5 million over 3 years to conduct the phase 2 study (NCT03556384). The objective of the study is to determine the efficacy at 6 months of temozolomide therapy in patients with the SDH-mutant/deficient subtype. Temozolomide is approved by the FDA to treat newly diagnosed glioblastoma multiforme and refractory anaplastic astrocytomas. It is not approved for the treatment of SDH-mutant/deficient GIST.
The FDA awarded the grants through the Orphan Products Clinical Trials Grants Program. Other orphan diseases receiving grants included glomerulopathy, gliomas, Fanconi anemia, sickle cell respiratory complications, Duchenne muscular dystrophy, HPV associated respiratory papillomatosis, refractory viral infections and T-cell immunodeficiency, oral cancer, retinoblastoma, cerebellar brain tumors, and acute myeloid leukemia.
FDA approves pembrolizumab for BCG-unresponsive NMIBC with CIS
The Food and Drug Administration has approved pembrolizumab (Keytruda) for the treatment of patients with Bacillus Calmette-Guérin (BCG)–unresponsive, high-risk, non–muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors, who are ineligible for or have elected not to undergo cystectomy.
The approval was based on response in a single-arm trial of 148 patients with high-risk NMIBC, 96 of whom had BCG-unresponsive CIS with or without papillary tumors, the FDA said in a statement.
The complete response rate in the 96 patients with high-risk BCG-unresponsive NMIBC with CIS was 41% (95% confidence interval, 31-51) and median response duration was 16.2 months.
The most common adverse reactions in patients who received pembrolizumab in the trial were fatigue, diarrhea, rash, pruritus, musculoskeletal pain, hematuria, cough, arthralgia, nausea, constipation, urinary tract infection, peripheral edema, hypothyroidism, and nasopharyngitis.
The recommended dose is 200 mg every 3 weeks, the FDA said.
The Food and Drug Administration has approved pembrolizumab (Keytruda) for the treatment of patients with Bacillus Calmette-Guérin (BCG)–unresponsive, high-risk, non–muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors, who are ineligible for or have elected not to undergo cystectomy.
The approval was based on response in a single-arm trial of 148 patients with high-risk NMIBC, 96 of whom had BCG-unresponsive CIS with or without papillary tumors, the FDA said in a statement.
The complete response rate in the 96 patients with high-risk BCG-unresponsive NMIBC with CIS was 41% (95% confidence interval, 31-51) and median response duration was 16.2 months.
The most common adverse reactions in patients who received pembrolizumab in the trial were fatigue, diarrhea, rash, pruritus, musculoskeletal pain, hematuria, cough, arthralgia, nausea, constipation, urinary tract infection, peripheral edema, hypothyroidism, and nasopharyngitis.
The recommended dose is 200 mg every 3 weeks, the FDA said.
The Food and Drug Administration has approved pembrolizumab (Keytruda) for the treatment of patients with Bacillus Calmette-Guérin (BCG)–unresponsive, high-risk, non–muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors, who are ineligible for or have elected not to undergo cystectomy.
The approval was based on response in a single-arm trial of 148 patients with high-risk NMIBC, 96 of whom had BCG-unresponsive CIS with or without papillary tumors, the FDA said in a statement.
The complete response rate in the 96 patients with high-risk BCG-unresponsive NMIBC with CIS was 41% (95% confidence interval, 31-51) and median response duration was 16.2 months.
The most common adverse reactions in patients who received pembrolizumab in the trial were fatigue, diarrhea, rash, pruritus, musculoskeletal pain, hematuria, cough, arthralgia, nausea, constipation, urinary tract infection, peripheral edema, hypothyroidism, and nasopharyngitis.
The recommended dose is 200 mg every 3 weeks, the FDA said.
Fast-acting, mealtime insulin aspart is approved for kids
making it the first fast-acting mealtime insulin injection that does not come with a premeal dosing recommendation, according to a release.
The injection is now available in various dosing options for both adult and pediatric patients with diabetes. Fast-acting mealtime insulin was approved in September 2017 for adults with type 1 or 2 disease, and in October 2019, it was approved for use in insulin pumps for adults.
The most recent approval was based on findings from the onset 7 trial, a 26-week, phase 3b, partially double-blind, treat-to-target trial that included 777 patients aged 1-18 years and demonstrated noninferiority to ordinary, non–fast-acting insulin aspart (Diabetes Care. 2019 Jul;42[7]:1255-62).
Removal of the premeal dosing requirement could help better manage mealtime insulin needs in children, according to the release from Novo Nordisk.
Use of the mealtime insulin injection comes with concerns of serious side effects, such as hypoglycemia, hypokalemia, serious allergic reactions, and heart failure. Common side effects can include skin problems (such as rash, itching, and swelling), injection-site reactions, and weight gain.
making it the first fast-acting mealtime insulin injection that does not come with a premeal dosing recommendation, according to a release.
The injection is now available in various dosing options for both adult and pediatric patients with diabetes. Fast-acting mealtime insulin was approved in September 2017 for adults with type 1 or 2 disease, and in October 2019, it was approved for use in insulin pumps for adults.
The most recent approval was based on findings from the onset 7 trial, a 26-week, phase 3b, partially double-blind, treat-to-target trial that included 777 patients aged 1-18 years and demonstrated noninferiority to ordinary, non–fast-acting insulin aspart (Diabetes Care. 2019 Jul;42[7]:1255-62).
Removal of the premeal dosing requirement could help better manage mealtime insulin needs in children, according to the release from Novo Nordisk.
Use of the mealtime insulin injection comes with concerns of serious side effects, such as hypoglycemia, hypokalemia, serious allergic reactions, and heart failure. Common side effects can include skin problems (such as rash, itching, and swelling), injection-site reactions, and weight gain.
making it the first fast-acting mealtime insulin injection that does not come with a premeal dosing recommendation, according to a release.
The injection is now available in various dosing options for both adult and pediatric patients with diabetes. Fast-acting mealtime insulin was approved in September 2017 for adults with type 1 or 2 disease, and in October 2019, it was approved for use in insulin pumps for adults.
The most recent approval was based on findings from the onset 7 trial, a 26-week, phase 3b, partially double-blind, treat-to-target trial that included 777 patients aged 1-18 years and demonstrated noninferiority to ordinary, non–fast-acting insulin aspart (Diabetes Care. 2019 Jul;42[7]:1255-62).
Removal of the premeal dosing requirement could help better manage mealtime insulin needs in children, according to the release from Novo Nordisk.
Use of the mealtime insulin injection comes with concerns of serious side effects, such as hypoglycemia, hypokalemia, serious allergic reactions, and heart failure. Common side effects can include skin problems (such as rash, itching, and swelling), injection-site reactions, and weight gain.