Accelerated Approval Withdrawal Offers Hints On New FDA Approach

Inside Health Policy, Luke Zarzecki, March 4, 2024

FDA’s decision to rescind accelerated approval of Oncopeptides AB’s multiple myeloma drug Pepaxto could offer a glimpse of potential policies to be included in a draft guidance due in June on accelerated approval rescissions, and poses unclear implications for other drugs as rare disease stakeholders call for the agency to step up use of the accelerated approval pathway.


FDA said in its statement on the decision the confirmatory study for Pepaxto did not confirm clinical benefit and the evidence did not show the drug to be safe or effective under conditions of use.

The rescission points to a problem Diana Zuckerman, president of the National Center for Health Research, sees with accelerated approval. Zuckerman told Inside Health Policy FDA’s new “’streamlined’ path to rescind accelerated approval hasn’t yet solved the basic problem with accelerated approval, which is that patients are being prescribed expensive drugs and biologics that are not proven to be safe and effective, and even after there is proof that patients are being harmed it can take years for FDA to get them off the market if the company doesn’t want to do so.”

With the Pepaxto approval rescinded, Zuckerman said the implications for other drugs remain unclear for a few reasons. “

1: FDA has known since 2021 that patients taking Pepaxto were more likely to die than patients using a different treatment.  #2: The company stopped selling Pepaxto in the U.S. in 2021, so FDA finally rescinding approval almost 3 years later is not saving any lives.  #3: It still took a long time to rescind FDA approval, even though an FDA Advisory Committee voted overwhelmingly that the drug was dangerous in 2022,” she wrote in an email.

Other treatments have also failed confirmatory trials but remain on the market. For example, a July 2023 letter from the Patient, Consumer, and Public Health Coalition raised concerns over the accelerated approval of Sarepta’s gene therapy for Duchenne muscular dystrophy Elevidys. That treatment failed its confirmatory trials. FDA is now considering expanding approval of Elevidys without an advisory committee meeting, the company announced recently.

The Coalition letter says biologics center director Peter Marks told the advisory committee to show flexibility by granting the approval of the drug.

“We consider this flexibility especially problematic because FDA had previously granted accelerated approval to three other Sarepta drugs, none of which have completed their confirmatory trials. In fact, the confirmatory trial for Exondys 51, which was approved in 2016, were due in November 2020 but instead was not even started until July 2020,” the letter says.


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