FDA issues alert about mortality risk with Pepaxto for multiple myeloma
The FDA alerted health care providers and patients about clinical trial results that showed increased mortality risk among patients with multiple myeloma treated with melphalan flufenamide.
The FDA in February granted accelerated approval to melphalan flufenamide (Pepaxto, Oncopeptides) in combination with dexamethasone for treatment of adults with relapsed or refractory multiple myeloma who received at least four prior lines of therapy and whose disease was refractory to at least one proteasome inhibitor, one immunomodulatory agent and one CD38-directed monoclonal antibody.
Under terms of the accelerated approval, the agency required Oncopeptides to conduct the randomized phase 3 OCEAN trial.
The confirmatory OCEAN trial compared the combination of melphalan flufenamide and low dexamethasone vs. pomalidomide (Pomalyst, Bristol Myers Squibb) and low-dose dexamethasone for patients with relapsed or refractory multiple myeloma who had received two to four prior lines of therapy and those who were resistant to lenalidomide (Revlimid, Bristol Myers Squibb) in their most recent line of therapy.
Results showed a detrimental effect on OS among melphalan flufenamide-treated patients, prompting FDA to require the manufacturer to suspend enrollment of the OCEAN trial and other ongoing trials of the agent.
FDA officials will continue to assess OCEAN trial results and may hold a public meeting to review safety findings and discuss the continued marketing of melphalan flufenamide, according to the alert.
Patients who are deriving clinical benefit from melphalan flufenamide in the OCEAN trial may continue treatment provided they sign a revised written informed consent.
The FDA alert encouraged health care providers to review patients’ progress on melphalan flufenamide and talk with them about the risks of continued administration.
Health care providers and patients are encouraged to report adverse events to FDA’s MedWatch Adverse Event Reporting program.
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