Akcea’s volanesorsen for familial chylomicronemia syndrome barely made it through advisory committee due to a rare but unpredictable and rapidly presenting adverse event that likely can’t be resolved with a REMS. Now what?
Many years ago, around the time when the US FDA was considering a “reevaluation” of the erythropoiesis-stimulating agents (ESAs) with a forthcoming advisory committee, the agency's dean of drug development Bob Temple was asked how the agency prepared for a panel meeting.
“I participate in a lot of these advisory committees," Temple said. "You have got to be careful. You don't want...
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Replimune's RP1 oncolytic immunotherapy became the third regenerative medicine to receive an FDA complete response letter this month as the agency appears to re-visit previous agreements about accelerated approval.
The 43 novel agents with user fee goal dates in the remainder of 2025 have fewer oncologics and infectious disease drugs than the first half of the year.
US FDA's 25 novel approvals in 2025 fell short of the agency's five-year average count, driven by a drop in drugs center approvals; 45 user fee goals remain in second half of year.
The transparency initiative collects CRLs already made public in approval packages on its drugs@fda site to post on the openFDA public portal.
The transparency initiative collects CRLs already made public in approval packages on its drugs@fda site to post on the openFDA public portal.
The US FDA will not be in its usual position as the first regulator to approve novel drugs, with only one of the six novel candidates on the July user fee calendar seeking its first approval worldwide in the US
This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.
