The FDA today issued a notice warning patients and healthcare professionals of a potentially increased risk of death with Pepaxto.
Pepaxto (melphalan flufenamide), used with dexamethasone to treat patients with multiple myeloma, demonstrated an increased risk of death in the OCEAN clinical trial. The FDA required that the manufacturer, Boston-based Oncopeptides (STO:ONCO) suspend enrollment for the trial.
Oncopeptides received accelerated FDA approval for Pepaxto on Feb. 26, then initiated the commercial launch of the drug on March 15. It touts the drug as the first anticancer peptide-drug conjugate approved for multiple myeloma.
Pepaxto links a peptide carrier to a cytotoxic agent, resulting in a lipophilic compound that helps to deliver the therapeutic into cells to leverage aminopeptidases that are overexpressed in multiple myeloma cells and cause the release of the cytotoxic agents. It is administered once a month in a 30-minute infusion process.
The company was required to conduct the OCEAN trial as a post-approval requirement under the accelerated approval program.
The OCEAN trial compared Pepaxto with low-dose dexamethosone to pomalidomide with low-dose dexamethasone in patients with relapsed or refractory (resistant) multiple myeloma following two to four lines of prior therapy and in patients who were resistant to lenalidomide in the last line of therapy, according to the FDA notice.
In addition to Oncopeptides’ trial, the FDA suspended enrollment in other ongoing Pepaxto clinical trials. Patients receiving clinical benefit from Pepaxto may continue treatment in the OCEAN trial provided that they are informed of the risks and sign a revised written informed consent.
The FDA said it will continue to evaluate the OCEAN trial results and may hold a future public meeting to discuss safety findings and to explore the continued marketing of Pepaxto.