FDA withdraws approval of cancer drug Pepaxto
The available evidence, FDA says, demonstrates that Pepaxto is not shown to be safe or effective under its conditions of use.
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The Food and Drug Administration has announced its final decision to withdraw approval of Pepaxto, which was approved for use in combination with dexamethasone to treat certain patients with multiple myeloma.
The agency determined that the drug's clinical benefit was not confirmed by a recent study conducted as a condition of accelerated approval. The available evidence, the FDA said, demonstrates that Pepaxto is not shown to be safe or effective under its conditions of use.
The final decision – which is effective immediately – was issued by FDA's Center for Biologics Evaluation and Research Director Dr. Peter Marks as the Commissioner's designee.
The FDA plans to publish a Federal Register notice regarding the decision and will remove Pepaxto from the Orange Book.
WHAT'S THE IMPACT?
This is the first time FDA has used the amended procedures for withdrawal of accelerated approval that were enacted in 2023, as part of the Food and Drug Omnibus Report Act of 2022 (FDORA).
In accordance with the new procedures, the FDA provided the drug's manufacturer, Oncopeptides, with a notice of proposed withdrawal of approval, an explanation for the proposed withdrawal and an opportunity for a meeting and a written appeal to the commissioner (or designee).
Oncopeptides submitted a written appeal and met with the Commissioner's designee. The decision is the FDA's response.
An Oncologic Drugs Advisory Committee (ODAC) was held on September 22, 2022, to discuss the results of an OCEAN confirmatory trial and the benefit-risk profile of Pepaxto. The ODAC voted 14 to 2 that the benefit-risk profile of melphalan flufenamide was not favorable for the currently indicated patient population.
THE LARGER TREND
A 2019 Tufts analysis found that cancer drugs account for 27% of all new drug approvals in the U.S. since 2010, a dramatic increase from the 4% share of the 1980s.
From 1980 through 2018, the FDA approved a total of 126 cancer drugs.
Pressure for even more oncology drugs is likely to continue, the analysis showed, given that there are still many cancers that are inadequately treated, or untreatable altogether.
Jeff Lagasse is editor of Healthcare Finance News.
Email: jlagasse@himss.org
Healthcare Finance News is a HIMSS Media publication.
