The US Food and Drug Administration (FDA) has announced its final decision to withdraw approval for Pepaxto (melphalan flufenamide), a drug initially approved for use in combination with dexamethasone to treat certain patients with multiple myeloma (MM). The decision stems from two key factors: (1) a confirmatory study, conducted as part of the conditions for accelerated approval, failed to confirm Pepaxto’s clinical benefit, and (2) the available evidence indicates that Pepaxto is neither safe nor effective under its intended conditions of use.
Initially granted accelerated approval by the FDA in February 2021, Pepaxto was prescribed for adult patients with relapsed or refractory multiple myeloma who had undergone at least four prior lines of therapy. The manufacturer, Oncopeptides AB, was obligated to conduct the OCEAN trial as a post-approval requirement. This trial revealed an overall survival risk ratio of 1.104 in the intention-to-treat patient population, signifying an increased risk of death. Consequently, the drug was delisted in October 2021.
The final decision was issued by Peter Marks, M.D., Ph.D., Director of the FDA’s Center for Biologics Evaluation and Research, acting as the Commissioner’s designee. The FDA intends to publish a Federal Register notice announcing the decision and will remove Pepaxto from the Orange Book. It is noted that Oncopeptides is not currently marketing Pepaxto in the U.S.
This action by the FDA underscores the agency’s commitment to ensuring the safety and efficacy of drugs on the market, particularly in the context of accelerated approval programs.- Flcube.com
