FDA withdraws approval for Pepaxto as a multiple myeloma treatment
Key takeaways:
- The FDA withdrew approval for a multiple myeloma treatment that was pulled from the market in October 2021.
- The agency determined the drug is not safe or effective under its conditions of use.
The FDA announced its final decision to rescind approval of melphalan flufenamide, citing safety concerns.
The agency approved melphalan flufenamide (Pepaxto, Oncopeptides) for use in combination with dexamethasone to treat certain patients with multiple myeloma in February 2021 under the Accelerated Approval Program.
A few months later, in July, the FDA alerted patients and health care providers about clinical trial results that showed an increased mortality risk among patients treated with the drug. In October, the manufacturer pulled the therapy from the market.
The decision, which is effective immediately, was issued by Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research. According to a press release, the FDA made the decision based on the following determinations: the “confirmatory study conducted as a condition of accelerated approval did not confirm” the drug’s clinical benefit and the available evidence indicates that it is not safe or effective under its conditions of use.
The release also states that this marks the first time the agency “has used the amended procedures for withdrawal of accelerated approval that were enacted in 2023,” as part of the 2022 Food and Drug Omnibus Report Act.
“In accordance with the new procedures, FDA provided 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),” according to the release. “Oncopeptides submitted a written appeal and met with the Commissioner’s designee. This decision is FDA’s response.”
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