Melphalan Flufenamide Is Withdrawn as Myeloma Treatment From U.S Markets
Posted: Friday, October 29, 2021
On October 22, Oncopeptides, the manufacturer of the first-in-class peptide-drug conjugate melphalan flufenamide (Pepaxto) withdrew the drug from the U.S. market. Melphalan flufenamide had previously received accelerated approval from the U.S. Food and Drug Administration (FDA) earlier this year.
The withdrawal decision was made after recent findings from the phase III OCEAN clinical trial studying melphalan flufenamide’s effectiveness in patients with relapsed or refractory multiple myeloma. The results demonstrated overall survival in the intent-to-treat population with a hazard ratio of 1.104. According to a press release from the company, after discussion with the FDA, “it has become evident that the FDA does not consider that the phase III OCEAN meets the criteria of a confirmatory study.” The FDA and Oncopeptides plan to work to make melphalan flufenamide accessible to patients who are currently using it.
Melphalan flufenamide is under consideration for a potential conditional marketing authorization for the European Medicines Agency. The approval decision is dependent on results from the phase II HORIZON trial, which is studying its use in the treatment of patients with relapsed or refractory multiple myeloma.