User login
FDA approves ibrutinib for previously treated MCL
The US Food and Drug Administration (FDA) has has granted accelerated approval for
ibrutinib (Imbruvica) to treat patients with mantle cell lymphoma (MCL) who have received at least 1 prior therapy.
Ibrutinib works by inhibiting the function of Bruton’s tyrosine kinase, a molecule that plays an important role in the survival of malignant B cells.
The drug showed promising results in the phase 2 PCYC-1104 trial, which was presented at ASH 2012 and published in NEJM in August.
The FDA granted ibrutinib breakthrough therapy designation because of these results and the life-threatening nature of MCL. Ibrutinib is the second drug with breakthrough therapy designation to receive FDA approval.
The FDA granted ibrutinib accelerated approval, rather than traditional approval, because the drug has not yet shown a clinical benefit. Accelerated approval of a drug is based
on a surrogate or intermediate endpoint—in this case, overall response rate—that is reasonably likely to
predict clinical benefit.
PCYC-1104 trial
The data published in NEJM included 111 patients who received ibrutinib at 560 mg daily in continuous, 28-day cycles until disease progression.
The
overall response rate was 68%, with a complete response rate of 21% and
a partial response rate of 47%. With an estimated median follow-up of
15.3 months, the estimated median response duration was 17.5 months.
The
estimated progression-free survival was 13.9 months, and the overall
survival was not reached. The estimated rate of overall survival was 58%
at 18 months.
Common nonhematologic adverse events included
diarrhea (50%), fatigue (41%), nausea (31%), peripheral edema (28%),
dyspnea (27%), constipation (25%), upper respiratory tract infection
(23%), vomiting (23%), and decreased appetite (21%). The most common
grade 3, 4, or 5 infection was pneumonia (6%).
Grade 3 and 4
hematologic adverse events included neutropenia (16%), thrombocytopenia
(11%), and anemia (10%). Grade 3 bleeding events occurred in 5 patients.
The “Warnings and Precautions” section of ibrutinib’s prescribing information notes that patients taking ibrutinib have experienced hemorrhage, fatal and non-fatal infections, myelosuppression, renal toxicity, second primary malignancies, and embryo-fetal toxicity.
For the full prescribing information, visit http://www.imbruvica.com/downloads/Prescribing_Information.pdf.
Ibrutinib is now commercially available. It is co-marketed by Pharmacyclics (based in Sunnyvale, California) and Janssen Biotech, Inc. (based in Raritan, New Jersey). 
The US Food and Drug Administration (FDA) has has granted accelerated approval for
ibrutinib (Imbruvica) to treat patients with mantle cell lymphoma (MCL) who have received at least 1 prior therapy.
Ibrutinib works by inhibiting the function of Bruton’s tyrosine kinase, a molecule that plays an important role in the survival of malignant B cells.
The drug showed promising results in the phase 2 PCYC-1104 trial, which was presented at ASH 2012 and published in NEJM in August.
The FDA granted ibrutinib breakthrough therapy designation because of these results and the life-threatening nature of MCL. Ibrutinib is the second drug with breakthrough therapy designation to receive FDA approval.
The FDA granted ibrutinib accelerated approval, rather than traditional approval, because the drug has not yet shown a clinical benefit. Accelerated approval of a drug is based
on a surrogate or intermediate endpoint—in this case, overall response rate—that is reasonably likely to
predict clinical benefit.
PCYC-1104 trial
The data published in NEJM included 111 patients who received ibrutinib at 560 mg daily in continuous, 28-day cycles until disease progression.
The
overall response rate was 68%, with a complete response rate of 21% and
a partial response rate of 47%. With an estimated median follow-up of
15.3 months, the estimated median response duration was 17.5 months.
The
estimated progression-free survival was 13.9 months, and the overall
survival was not reached. The estimated rate of overall survival was 58%
at 18 months.
Common nonhematologic adverse events included
diarrhea (50%), fatigue (41%), nausea (31%), peripheral edema (28%),
dyspnea (27%), constipation (25%), upper respiratory tract infection
(23%), vomiting (23%), and decreased appetite (21%). The most common
grade 3, 4, or 5 infection was pneumonia (6%).
Grade 3 and 4
hematologic adverse events included neutropenia (16%), thrombocytopenia
(11%), and anemia (10%). Grade 3 bleeding events occurred in 5 patients.
The “Warnings and Precautions” section of ibrutinib’s prescribing information notes that patients taking ibrutinib have experienced hemorrhage, fatal and non-fatal infections, myelosuppression, renal toxicity, second primary malignancies, and embryo-fetal toxicity.
For the full prescribing information, visit http://www.imbruvica.com/downloads/Prescribing_Information.pdf.
Ibrutinib is now commercially available. It is co-marketed by Pharmacyclics (based in Sunnyvale, California) and Janssen Biotech, Inc. (based in Raritan, New Jersey).
The US Food and Drug Administration (FDA) has has granted accelerated approval for
ibrutinib (Imbruvica) to treat patients with mantle cell lymphoma (MCL) who have received at least 1 prior therapy.
Ibrutinib works by inhibiting the function of Bruton’s tyrosine kinase, a molecule that plays an important role in the survival of malignant B cells.
The drug showed promising results in the phase 2 PCYC-1104 trial, which was presented at ASH 2012 and published in NEJM in August.
The FDA granted ibrutinib breakthrough therapy designation because of these results and the life-threatening nature of MCL. Ibrutinib is the second drug with breakthrough therapy designation to receive FDA approval.
The FDA granted ibrutinib accelerated approval, rather than traditional approval, because the drug has not yet shown a clinical benefit. Accelerated approval of a drug is based
on a surrogate or intermediate endpoint—in this case, overall response rate—that is reasonably likely to
predict clinical benefit.
PCYC-1104 trial
The data published in NEJM included 111 patients who received ibrutinib at 560 mg daily in continuous, 28-day cycles until disease progression.
The
overall response rate was 68%, with a complete response rate of 21% and
a partial response rate of 47%. With an estimated median follow-up of
15.3 months, the estimated median response duration was 17.5 months.
The
estimated progression-free survival was 13.9 months, and the overall
survival was not reached. The estimated rate of overall survival was 58%
at 18 months.
Common nonhematologic adverse events included
diarrhea (50%), fatigue (41%), nausea (31%), peripheral edema (28%),
dyspnea (27%), constipation (25%), upper respiratory tract infection
(23%), vomiting (23%), and decreased appetite (21%). The most common
grade 3, 4, or 5 infection was pneumonia (6%).
Grade 3 and 4
hematologic adverse events included neutropenia (16%), thrombocytopenia
(11%), and anemia (10%). Grade 3 bleeding events occurred in 5 patients.
The “Warnings and Precautions” section of ibrutinib’s prescribing information notes that patients taking ibrutinib have experienced hemorrhage, fatal and non-fatal infections, myelosuppression, renal toxicity, second primary malignancies, and embryo-fetal toxicity.
For the full prescribing information, visit http://www.imbruvica.com/downloads/Prescribing_Information.pdf.
Ibrutinib is now commercially available. It is co-marketed by Pharmacyclics (based in Sunnyvale, California) and Janssen Biotech, Inc. (based in Raritan, New Jersey).
Fraud, errors were behind delay of apixaban approval, report shows
Credit: Esther Dyson
Medication mistakes, reporting errors, and record changes are what led the US Food and Drug Administration (FDA) to delay approval of the anticoagulant apixaban (Eliquis), according to a report in Pharmaceutical Approvals Monthly.
The report reveals that a number of patients enrolled on the ARISTOTLE trial received the wrong medication or the wrong dose, some serious adverse events went unreported, and employees who worked at trial sites in China altered records to cover up noncompliance with “good clinical practice.”
Apixaban won FDA approval last December as prophylaxis for stroke and systemic embolism in patients with nonvalvular atrial fibrillation. And that approval was based on results of the ARISTOTLE trial.
Prior to the approval, the FDA twice rejected a new drug application filed by apixaban’s developers, Pfizer and Bristol-Myers Squibb. But the agency’s reasons were not immediately made public.
Now, the Pharmaceutical Approvals Monthly report and FDA documents show that “data irregularities” were behind the decision.
In January 2012, Bristol-Myers Squibb notified the FDA of the cover-up attempt that occurred at (at least) 1 trial site in China. The company had learned that its senior manager at a site in Shanghai and an employee from a contract research organization had altered source records to conceal good clinical practice violations. (The employees were subsequently fired.)
According to FDA documents, the cover-up included failure to report 4 potential adverse events, late reports on 3 other events, and the omission of 3 patient outcomes. In addition, there were errors in patient names and dates, some Chinese and English records didn’t match up, and some patient records disappeared before a site visit by FDA inspectors.
Only 35 patients enrolled in the ARISTOTLE trial were treated at the Shanghai site, but the employees who perpetrated the fraud had also worked at 24 of the 36 trial sites in China.
So the FDA performed analyses excluding data from the Shanghai site alone, from the 24 sites where the employees worked, and from all 36 sites in China. And they found that apixaban’s efficacy still held up.
However, there was still the issue of ARISTOTLE participants receiving the wrong medication or the wrong dose. According to Bristol-Myers Squibb, the trial’s double-blind design allowed for dispensing errors.
Initially, the company said this meant that 7.3% of patients who were set to receive apixaban and 1.2% of patients who were set to receive warfarin may have received the wrong drug or dose at some point during the study. However, after additional review, the company said those percentages were likely much lower.
Regardless of the actual percentages, the FDA said this information suggests a pattern of inadequate oversight. And an independent review by FDA medical team leader Thomas Marciniak appears to support that statement. In addition to the aforementioned errors, his review revealed records of doctor visits taking place after patients’ deaths.
In spite of the errors and the fraud, the FDA said it remained convinced of apixaban’s efficacy. So the agency decided to approve the drug and leave out any mention of the data irregularities on the drug’s label.
Credit: Esther Dyson
Medication mistakes, reporting errors, and record changes are what led the US Food and Drug Administration (FDA) to delay approval of the anticoagulant apixaban (Eliquis), according to a report in Pharmaceutical Approvals Monthly.
The report reveals that a number of patients enrolled on the ARISTOTLE trial received the wrong medication or the wrong dose, some serious adverse events went unreported, and employees who worked at trial sites in China altered records to cover up noncompliance with “good clinical practice.”
Apixaban won FDA approval last December as prophylaxis for stroke and systemic embolism in patients with nonvalvular atrial fibrillation. And that approval was based on results of the ARISTOTLE trial.
Prior to the approval, the FDA twice rejected a new drug application filed by apixaban’s developers, Pfizer and Bristol-Myers Squibb. But the agency’s reasons were not immediately made public.
Now, the Pharmaceutical Approvals Monthly report and FDA documents show that “data irregularities” were behind the decision.
In January 2012, Bristol-Myers Squibb notified the FDA of the cover-up attempt that occurred at (at least) 1 trial site in China. The company had learned that its senior manager at a site in Shanghai and an employee from a contract research organization had altered source records to conceal good clinical practice violations. (The employees were subsequently fired.)
According to FDA documents, the cover-up included failure to report 4 potential adverse events, late reports on 3 other events, and the omission of 3 patient outcomes. In addition, there were errors in patient names and dates, some Chinese and English records didn’t match up, and some patient records disappeared before a site visit by FDA inspectors.
Only 35 patients enrolled in the ARISTOTLE trial were treated at the Shanghai site, but the employees who perpetrated the fraud had also worked at 24 of the 36 trial sites in China.
So the FDA performed analyses excluding data from the Shanghai site alone, from the 24 sites where the employees worked, and from all 36 sites in China. And they found that apixaban’s efficacy still held up.
However, there was still the issue of ARISTOTLE participants receiving the wrong medication or the wrong dose. According to Bristol-Myers Squibb, the trial’s double-blind design allowed for dispensing errors.
Initially, the company said this meant that 7.3% of patients who were set to receive apixaban and 1.2% of patients who were set to receive warfarin may have received the wrong drug or dose at some point during the study. However, after additional review, the company said those percentages were likely much lower.
Regardless of the actual percentages, the FDA said this information suggests a pattern of inadequate oversight. And an independent review by FDA medical team leader Thomas Marciniak appears to support that statement. In addition to the aforementioned errors, his review revealed records of doctor visits taking place after patients’ deaths.
In spite of the errors and the fraud, the FDA said it remained convinced of apixaban’s efficacy. So the agency decided to approve the drug and leave out any mention of the data irregularities on the drug’s label.
Credit: Esther Dyson
Medication mistakes, reporting errors, and record changes are what led the US Food and Drug Administration (FDA) to delay approval of the anticoagulant apixaban (Eliquis), according to a report in Pharmaceutical Approvals Monthly.
The report reveals that a number of patients enrolled on the ARISTOTLE trial received the wrong medication or the wrong dose, some serious adverse events went unreported, and employees who worked at trial sites in China altered records to cover up noncompliance with “good clinical practice.”
Apixaban won FDA approval last December as prophylaxis for stroke and systemic embolism in patients with nonvalvular atrial fibrillation. And that approval was based on results of the ARISTOTLE trial.
Prior to the approval, the FDA twice rejected a new drug application filed by apixaban’s developers, Pfizer and Bristol-Myers Squibb. But the agency’s reasons were not immediately made public.
Now, the Pharmaceutical Approvals Monthly report and FDA documents show that “data irregularities” were behind the decision.
In January 2012, Bristol-Myers Squibb notified the FDA of the cover-up attempt that occurred at (at least) 1 trial site in China. The company had learned that its senior manager at a site in Shanghai and an employee from a contract research organization had altered source records to conceal good clinical practice violations. (The employees were subsequently fired.)
According to FDA documents, the cover-up included failure to report 4 potential adverse events, late reports on 3 other events, and the omission of 3 patient outcomes. In addition, there were errors in patient names and dates, some Chinese and English records didn’t match up, and some patient records disappeared before a site visit by FDA inspectors.
Only 35 patients enrolled in the ARISTOTLE trial were treated at the Shanghai site, but the employees who perpetrated the fraud had also worked at 24 of the 36 trial sites in China.
So the FDA performed analyses excluding data from the Shanghai site alone, from the 24 sites where the employees worked, and from all 36 sites in China. And they found that apixaban’s efficacy still held up.
However, there was still the issue of ARISTOTLE participants receiving the wrong medication or the wrong dose. According to Bristol-Myers Squibb, the trial’s double-blind design allowed for dispensing errors.
Initially, the company said this meant that 7.3% of patients who were set to receive apixaban and 1.2% of patients who were set to receive warfarin may have received the wrong drug or dose at some point during the study. However, after additional review, the company said those percentages were likely much lower.
Regardless of the actual percentages, the FDA said this information suggests a pattern of inadequate oversight. And an independent review by FDA medical team leader Thomas Marciniak appears to support that statement. In addition to the aforementioned errors, his review revealed records of doctor visits taking place after patients’ deaths.
In spite of the errors and the fraud, the FDA said it remained convinced of apixaban’s efficacy. So the agency decided to approve the drug and leave out any mention of the data irregularities on the drug’s label.
Company suspends enrollment in drug trials
Credit: Esther Dyson
After 2 deaths among patients receiving the BCL-2 inhibitor ABT-199, the company developing the drug has suspended enrollment in 5 trials and stopped dose-escalation of the drug.
The patients died of tumor lysis syndrome, a complication that likely stems from the drug’s potency, according to Tracy Sorrentino, a spokeswoman for the company, AbbVie.
Research has suggested the risk of tumor lysis syndrome might be eliminated by altering the dose of ABT-199, Sorrentino said.
But until that is confirmed, AbbVie has stopped dose-escalation in patients receiving ABT-199 and voluntarily suspended enrollment in phase 1 trials of the drug.
The trials are testing ABT-199, both alone and in combination, as a treatment for chronic lymphocytic leukemia, non-Hodgkin lymphoma, and small lymphocytic lymphoma.
Though enrollment has stopped for these trials, dosing of active patients in ABT-199 trials will continue. In addition, a study testing ABT-199 in women with systemic lupus erythematosus is still enrolling patients.
Sorrentino said AbbVie has “every expectation” the suspended enrollment is temporary, and refining the dose of ABT-199 may eliminate the problem. In fact, the company is still planning to begin phase 3 trials of the drug later this year.
Credit: Esther Dyson
After 2 deaths among patients receiving the BCL-2 inhibitor ABT-199, the company developing the drug has suspended enrollment in 5 trials and stopped dose-escalation of the drug.
The patients died of tumor lysis syndrome, a complication that likely stems from the drug’s potency, according to Tracy Sorrentino, a spokeswoman for the company, AbbVie.
Research has suggested the risk of tumor lysis syndrome might be eliminated by altering the dose of ABT-199, Sorrentino said.
But until that is confirmed, AbbVie has stopped dose-escalation in patients receiving ABT-199 and voluntarily suspended enrollment in phase 1 trials of the drug.
The trials are testing ABT-199, both alone and in combination, as a treatment for chronic lymphocytic leukemia, non-Hodgkin lymphoma, and small lymphocytic lymphoma.
Though enrollment has stopped for these trials, dosing of active patients in ABT-199 trials will continue. In addition, a study testing ABT-199 in women with systemic lupus erythematosus is still enrolling patients.
Sorrentino said AbbVie has “every expectation” the suspended enrollment is temporary, and refining the dose of ABT-199 may eliminate the problem. In fact, the company is still planning to begin phase 3 trials of the drug later this year.
Credit: Esther Dyson
After 2 deaths among patients receiving the BCL-2 inhibitor ABT-199, the company developing the drug has suspended enrollment in 5 trials and stopped dose-escalation of the drug.
The patients died of tumor lysis syndrome, a complication that likely stems from the drug’s potency, according to Tracy Sorrentino, a spokeswoman for the company, AbbVie.
Research has suggested the risk of tumor lysis syndrome might be eliminated by altering the dose of ABT-199, Sorrentino said.
But until that is confirmed, AbbVie has stopped dose-escalation in patients receiving ABT-199 and voluntarily suspended enrollment in phase 1 trials of the drug.
The trials are testing ABT-199, both alone and in combination, as a treatment for chronic lymphocytic leukemia, non-Hodgkin lymphoma, and small lymphocytic lymphoma.
Though enrollment has stopped for these trials, dosing of active patients in ABT-199 trials will continue. In addition, a study testing ABT-199 in women with systemic lupus erythematosus is still enrolling patients.
Sorrentino said AbbVie has “every expectation” the suspended enrollment is temporary, and refining the dose of ABT-199 may eliminate the problem. In fact, the company is still planning to begin phase 3 trials of the drug later this year.
FDA approves pomalidomide for MM
Credit: Steven Harbour
The US Food and Drug Administration (FDA) has granted accelerated approval for the immunomodulatory agent pomalidomide (Pomalyst) to treat patients with advanced multiple myeloma (MM).
Continued FDA approval for the drug may be contingent upon verification and description of clinical benefit in confirmatory trials.
Pomalidomide is intended for use in combination with dexamethasone to treat MM patients who have received at least 2 prior
therapies (including lenalidomide and a proteasome inhibitor) and who experienced progression within 60 days of their last treatment.
Pomalidomide has demonstrated some efficacy in this patient population in a number of studies.
In a study published in Blood last year (PG Richardson et al.), pomalidomide elicited responses in MM patients who were refractory to lenalidomide, bortezomib, or both drugs.
In a study presented at ASH 2011 (abstract 634), pomalidomide did not fare as well when given alone to patients with refractory MM. However, combining the drug with low-dose dexamethasone significantly improved responses.
A study presented at ASH 2012 (LBA-6) built upon those findings, showing that pomalidomide plus low-dose dexamethasone was superior to high-dose dexamethasone in MM patients who were refractory to lenalidomide and bortezomib.
Common side effects observed with pomalidomide include neutropenia, anemia, thrombocytopenia, fatigue, weakness, constipation, diarrhea, upper respiratory tract infections, back pain, and fever.
In addition, pomalidomide has been shown to cause venous thromboembolism, as well as severe, life-threatening birth defects in pregnant women. The drug carries a boxed warning alerting patients and healthcare professionals to both of these risks.
Because of the embryo-fetal risk, pomalidomide is available only through the Pomalyst Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribers must be certified with the program by enrolling and complying with the REMS requirements.
Patients must sign a patient-physician agreement form and comply with the REMS requirements. In particular, female patients who are not pregnant but can become pregnant must comply with the pregnancy testing and contraception requirements, and males must comply with contraception requirements.
Pharmacies must be certified with the Pomalyst REMS Program, must only dispense the drug to patients who are authorized to receive it, and must comply with REMS requirements. Both lenalidomide and thalidomide have similar REMS.
Pomalidomide is marketed by Celgene, which is based in Summit, New Jersey.
Credit: Steven Harbour
The US Food and Drug Administration (FDA) has granted accelerated approval for the immunomodulatory agent pomalidomide (Pomalyst) to treat patients with advanced multiple myeloma (MM).
Continued FDA approval for the drug may be contingent upon verification and description of clinical benefit in confirmatory trials.
Pomalidomide is intended for use in combination with dexamethasone to treat MM patients who have received at least 2 prior
therapies (including lenalidomide and a proteasome inhibitor) and who experienced progression within 60 days of their last treatment.
Pomalidomide has demonstrated some efficacy in this patient population in a number of studies.
In a study published in Blood last year (PG Richardson et al.), pomalidomide elicited responses in MM patients who were refractory to lenalidomide, bortezomib, or both drugs.
In a study presented at ASH 2011 (abstract 634), pomalidomide did not fare as well when given alone to patients with refractory MM. However, combining the drug with low-dose dexamethasone significantly improved responses.
A study presented at ASH 2012 (LBA-6) built upon those findings, showing that pomalidomide plus low-dose dexamethasone was superior to high-dose dexamethasone in MM patients who were refractory to lenalidomide and bortezomib.
Common side effects observed with pomalidomide include neutropenia, anemia, thrombocytopenia, fatigue, weakness, constipation, diarrhea, upper respiratory tract infections, back pain, and fever.
In addition, pomalidomide has been shown to cause venous thromboembolism, as well as severe, life-threatening birth defects in pregnant women. The drug carries a boxed warning alerting patients and healthcare professionals to both of these risks.
Because of the embryo-fetal risk, pomalidomide is available only through the Pomalyst Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribers must be certified with the program by enrolling and complying with the REMS requirements.
Patients must sign a patient-physician agreement form and comply with the REMS requirements. In particular, female patients who are not pregnant but can become pregnant must comply with the pregnancy testing and contraception requirements, and males must comply with contraception requirements.
Pharmacies must be certified with the Pomalyst REMS Program, must only dispense the drug to patients who are authorized to receive it, and must comply with REMS requirements. Both lenalidomide and thalidomide have similar REMS.
Pomalidomide is marketed by Celgene, which is based in Summit, New Jersey.
Credit: Steven Harbour
The US Food and Drug Administration (FDA) has granted accelerated approval for the immunomodulatory agent pomalidomide (Pomalyst) to treat patients with advanced multiple myeloma (MM).
Continued FDA approval for the drug may be contingent upon verification and description of clinical benefit in confirmatory trials.
Pomalidomide is intended for use in combination with dexamethasone to treat MM patients who have received at least 2 prior
therapies (including lenalidomide and a proteasome inhibitor) and who experienced progression within 60 days of their last treatment.
Pomalidomide has demonstrated some efficacy in this patient population in a number of studies.
In a study published in Blood last year (PG Richardson et al.), pomalidomide elicited responses in MM patients who were refractory to lenalidomide, bortezomib, or both drugs.
In a study presented at ASH 2011 (abstract 634), pomalidomide did not fare as well when given alone to patients with refractory MM. However, combining the drug with low-dose dexamethasone significantly improved responses.
A study presented at ASH 2012 (LBA-6) built upon those findings, showing that pomalidomide plus low-dose dexamethasone was superior to high-dose dexamethasone in MM patients who were refractory to lenalidomide and bortezomib.
Common side effects observed with pomalidomide include neutropenia, anemia, thrombocytopenia, fatigue, weakness, constipation, diarrhea, upper respiratory tract infections, back pain, and fever.
In addition, pomalidomide has been shown to cause venous thromboembolism, as well as severe, life-threatening birth defects in pregnant women. The drug carries a boxed warning alerting patients and healthcare professionals to both of these risks.
Because of the embryo-fetal risk, pomalidomide is available only through the Pomalyst Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribers must be certified with the program by enrolling and complying with the REMS requirements.
Patients must sign a patient-physician agreement form and comply with the REMS requirements. In particular, female patients who are not pregnant but can become pregnant must comply with the pregnancy testing and contraception requirements, and males must comply with contraception requirements.
Pharmacies must be certified with the Pomalyst REMS Program, must only dispense the drug to patients who are authorized to receive it, and must comply with REMS requirements. Both lenalidomide and thalidomide have similar REMS.
Pomalidomide is marketed by Celgene, which is based in Summit, New Jersey.
FDA approves drugs faster than EMA, Health Canada
The FDA generally approves drugs faster than its Canadian and European counterparts, according to a study published in this week’s edition of NEJM.
The researchers say these results refute criticisms that the drug approval process in the US is slow and that agencies in other countries tend to approve new therapies first.
“The perception that the FDA is too slow implies that sick patients are waiting unnecessarily for regulators to complete their review of new drug applications,” said lead study author Nicholas Downing, a medical student at Yale University.
He and his colleagues decided to conduct this study because there have been no recent comparisons of the FDA’s review speed with that of agencies in other countries.
So the researchers reviewed drug approval decisions made by the FDA, Health Canada, and the European Medicines Agency (EMA) between 2001 and 2010. The team said they chose Health Canada and the EMA as comparisons because these agencies face similar pressures to approve new drugs quickly while ensuring they don’t put patients at risk.
The investigators studied each regulator’s database of drug approvals to identify novel therapeutics, as well as the timing of key regulatory events. They then calculated each agency’s review speed.
The median total time to review a new drug application was 322 days at the FDA, 366 days at the EMA, and 393 days at Health Canada.
“Among the subsample of drugs approved for all 3 regulators, the FDA’s reviews were over 3 months faster than those of the EMA or Health Canada,” Downing said. “The total review time at the FDA was faster than EMA, despite the FDA’s far higher proportion of applications requiring multiple regulatory reviews.”
The researchers also found that, during the review period, the FDA approved 225 new drugs, the EMA approved 186, and Health Canada approved 99. Additionally, of the therapies that have been approved by all 3 agencies, most drugs were first approved in the US.
“[W]e found that 64% of medicines approved in both the US and in Europe were approved for US patients first,” Downing said. “And 86% of medicines approved in both the US and Canada were also approved first in the US.”
Downing and his colleagues noted that this study has 2 key limitations. First, the researchers didn’t account for drugs that were ultimately rejected, as the regulatory agencies don’t release review times for drugs that are never approved. However, the team also pointed out that the FDA approves more than 80% of its applications, so the exclusion may not have made much of an impact.
Secondly, the study included only new molecular entities and original biologic agents. In order to get a more accurate reading on the regulatory review process, research would need to evaluate the review of generic drugs, reformulated drugs, combination therapies, and medical devices.
The FDA generally approves drugs faster than its Canadian and European counterparts, according to a study published in this week’s edition of NEJM.
The researchers say these results refute criticisms that the drug approval process in the US is slow and that agencies in other countries tend to approve new therapies first.
“The perception that the FDA is too slow implies that sick patients are waiting unnecessarily for regulators to complete their review of new drug applications,” said lead study author Nicholas Downing, a medical student at Yale University.
He and his colleagues decided to conduct this study because there have been no recent comparisons of the FDA’s review speed with that of agencies in other countries.
So the researchers reviewed drug approval decisions made by the FDA, Health Canada, and the European Medicines Agency (EMA) between 2001 and 2010. The team said they chose Health Canada and the EMA as comparisons because these agencies face similar pressures to approve new drugs quickly while ensuring they don’t put patients at risk.
The investigators studied each regulator’s database of drug approvals to identify novel therapeutics, as well as the timing of key regulatory events. They then calculated each agency’s review speed.
The median total time to review a new drug application was 322 days at the FDA, 366 days at the EMA, and 393 days at Health Canada.
“Among the subsample of drugs approved for all 3 regulators, the FDA’s reviews were over 3 months faster than those of the EMA or Health Canada,” Downing said. “The total review time at the FDA was faster than EMA, despite the FDA’s far higher proportion of applications requiring multiple regulatory reviews.”
The researchers also found that, during the review period, the FDA approved 225 new drugs, the EMA approved 186, and Health Canada approved 99. Additionally, of the therapies that have been approved by all 3 agencies, most drugs were first approved in the US.
“[W]e found that 64% of medicines approved in both the US and in Europe were approved for US patients first,” Downing said. “And 86% of medicines approved in both the US and Canada were also approved first in the US.”
Downing and his colleagues noted that this study has 2 key limitations. First, the researchers didn’t account for drugs that were ultimately rejected, as the regulatory agencies don’t release review times for drugs that are never approved. However, the team also pointed out that the FDA approves more than 80% of its applications, so the exclusion may not have made much of an impact.
Secondly, the study included only new molecular entities and original biologic agents. In order to get a more accurate reading on the regulatory review process, research would need to evaluate the review of generic drugs, reformulated drugs, combination therapies, and medical devices.
The FDA generally approves drugs faster than its Canadian and European counterparts, according to a study published in this week’s edition of NEJM.
The researchers say these results refute criticisms that the drug approval process in the US is slow and that agencies in other countries tend to approve new therapies first.
“The perception that the FDA is too slow implies that sick patients are waiting unnecessarily for regulators to complete their review of new drug applications,” said lead study author Nicholas Downing, a medical student at Yale University.
He and his colleagues decided to conduct this study because there have been no recent comparisons of the FDA’s review speed with that of agencies in other countries.
So the researchers reviewed drug approval decisions made by the FDA, Health Canada, and the European Medicines Agency (EMA) between 2001 and 2010. The team said they chose Health Canada and the EMA as comparisons because these agencies face similar pressures to approve new drugs quickly while ensuring they don’t put patients at risk.
The investigators studied each regulator’s database of drug approvals to identify novel therapeutics, as well as the timing of key regulatory events. They then calculated each agency’s review speed.
The median total time to review a new drug application was 322 days at the FDA, 366 days at the EMA, and 393 days at Health Canada.
“Among the subsample of drugs approved for all 3 regulators, the FDA’s reviews were over 3 months faster than those of the EMA or Health Canada,” Downing said. “The total review time at the FDA was faster than EMA, despite the FDA’s far higher proportion of applications requiring multiple regulatory reviews.”
The researchers also found that, during the review period, the FDA approved 225 new drugs, the EMA approved 186, and Health Canada approved 99. Additionally, of the therapies that have been approved by all 3 agencies, most drugs were first approved in the US.
“[W]e found that 64% of medicines approved in both the US and in Europe were approved for US patients first,” Downing said. “And 86% of medicines approved in both the US and Canada were also approved first in the US.”
Downing and his colleagues noted that this study has 2 key limitations. First, the researchers didn’t account for drugs that were ultimately rejected, as the regulatory agencies don’t release review times for drugs that are never approved. However, the team also pointed out that the FDA approves more than 80% of its applications, so the exclusion may not have made much of an impact.
Secondly, the study included only new molecular entities and original biologic agents. In order to get a more accurate reading on the regulatory review process, research would need to evaluate the review of generic drugs, reformulated drugs, combination therapies, and medical devices.
Eculizumab gets accelerated approval for aHUS
The US Food and Drug Administration (FDA) has granted accelerated approval for eculizumab (Soliris) to treat patients with atypical hemolytic uremic syndrome (aHUS).
This rare and chronic disease can lead to renal failure and is associated with an increased risk of death and stroke. It accounts for 5% to 10% of all cases of hemolytic uremic syndrome and disproportionately affects children.
Eculizumab is a targeted therapy that works by inhibiting proteins that play a role in aHUS. The FDA granted accelerated approval for eculizumab based on data suggesting the drug likely confers a clinical benefit for patients with aHUS.
The FDA’s Accelerated Approval Program allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need based on a surrogate endpoint that is thought to predict clinical benefit. The makers of eculizumab, Alexion Pharmaceuticals, are still required to conduct research to confirm the anticipated clinical benefit.
If this research indicates that eculizumab does provide a clinical benefit, the FDA will grant traditional approval for the drug. If research suggests eculizumab does not provide a clinical benefit, the FDA has regulatory procedures in place that could lead to removing the drug from the market.
There are no other FDA-approved treatments for aHUS. The safety and efficacy of the current standard treatment, plasma therapy (plasma exchange or fresh frozen plasma infusion), have not been studied in well-controlled trials.
Researchers have examined the safety and efficacy of eculizumab in 2 single-arm trials of 37 adult and adolescent patients with aHUS and 1 retrospective study of 19 pediatric and 11 adult patients with aHUS.
Patients treated with eculizumab in these studies experienced a favorable improvement in kidney function, including elimination of the requirement for dialysis in several patients who did not respond to plasma therapy.
Patients treated with eculizumab also exhibited improvement in platelet counts and other blood parameters that correlate with aHUS disease activity.
The most common side effects included hypertension, diarrhea, headache, anemia, vomiting, nausea, upper respiratory and urinary tract infections, and leukopenia.
Eculizumab will continue to be available only through a restricted program. Prescribers must enroll in a registration program and provide a medication guide to patients who receive the drug.
Eculizumab is marketed as Soliris by Alexion Pharmaceuticals, located in Cheshire, Connecticut. 
The US Food and Drug Administration (FDA) has granted accelerated approval for eculizumab (Soliris) to treat patients with atypical hemolytic uremic syndrome (aHUS).
This rare and chronic disease can lead to renal failure and is associated with an increased risk of death and stroke. It accounts for 5% to 10% of all cases of hemolytic uremic syndrome and disproportionately affects children.
Eculizumab is a targeted therapy that works by inhibiting proteins that play a role in aHUS. The FDA granted accelerated approval for eculizumab based on data suggesting the drug likely confers a clinical benefit for patients with aHUS.
The FDA’s Accelerated Approval Program allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need based on a surrogate endpoint that is thought to predict clinical benefit. The makers of eculizumab, Alexion Pharmaceuticals, are still required to conduct research to confirm the anticipated clinical benefit.
If this research indicates that eculizumab does provide a clinical benefit, the FDA will grant traditional approval for the drug. If research suggests eculizumab does not provide a clinical benefit, the FDA has regulatory procedures in place that could lead to removing the drug from the market.
There are no other FDA-approved treatments for aHUS. The safety and efficacy of the current standard treatment, plasma therapy (plasma exchange or fresh frozen plasma infusion), have not been studied in well-controlled trials.
Researchers have examined the safety and efficacy of eculizumab in 2 single-arm trials of 37 adult and adolescent patients with aHUS and 1 retrospective study of 19 pediatric and 11 adult patients with aHUS.
Patients treated with eculizumab in these studies experienced a favorable improvement in kidney function, including elimination of the requirement for dialysis in several patients who did not respond to plasma therapy.
Patients treated with eculizumab also exhibited improvement in platelet counts and other blood parameters that correlate with aHUS disease activity.
The most common side effects included hypertension, diarrhea, headache, anemia, vomiting, nausea, upper respiratory and urinary tract infections, and leukopenia.
Eculizumab will continue to be available only through a restricted program. Prescribers must enroll in a registration program and provide a medication guide to patients who receive the drug.
Eculizumab is marketed as Soliris by Alexion Pharmaceuticals, located in Cheshire, Connecticut.
The US Food and Drug Administration (FDA) has granted accelerated approval for eculizumab (Soliris) to treat patients with atypical hemolytic uremic syndrome (aHUS).
This rare and chronic disease can lead to renal failure and is associated with an increased risk of death and stroke. It accounts for 5% to 10% of all cases of hemolytic uremic syndrome and disproportionately affects children.
Eculizumab is a targeted therapy that works by inhibiting proteins that play a role in aHUS. The FDA granted accelerated approval for eculizumab based on data suggesting the drug likely confers a clinical benefit for patients with aHUS.
The FDA’s Accelerated Approval Program allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need based on a surrogate endpoint that is thought to predict clinical benefit. The makers of eculizumab, Alexion Pharmaceuticals, are still required to conduct research to confirm the anticipated clinical benefit.
If this research indicates that eculizumab does provide a clinical benefit, the FDA will grant traditional approval for the drug. If research suggests eculizumab does not provide a clinical benefit, the FDA has regulatory procedures in place that could lead to removing the drug from the market.
There are no other FDA-approved treatments for aHUS. The safety and efficacy of the current standard treatment, plasma therapy (plasma exchange or fresh frozen plasma infusion), have not been studied in well-controlled trials.
Researchers have examined the safety and efficacy of eculizumab in 2 single-arm trials of 37 adult and adolescent patients with aHUS and 1 retrospective study of 19 pediatric and 11 adult patients with aHUS.
Patients treated with eculizumab in these studies experienced a favorable improvement in kidney function, including elimination of the requirement for dialysis in several patients who did not respond to plasma therapy.
Patients treated with eculizumab also exhibited improvement in platelet counts and other blood parameters that correlate with aHUS disease activity.
The most common side effects included hypertension, diarrhea, headache, anemia, vomiting, nausea, upper respiratory and urinary tract infections, and leukopenia.
Eculizumab will continue to be available only through a restricted program. Prescribers must enroll in a registration program and provide a medication guide to patients who receive the drug.
Eculizumab is marketed as Soliris by Alexion Pharmaceuticals, located in Cheshire, Connecticut.