Oncopeptides’ Pepaxto Appeal Falls Flat in CDER Rejection

September 19, 2023

In the latest in an ongoing dispute, CDER has strongly rejected Oncopeptides’ appeal of the agency’s attempt to request the drugmaker withdraw its multiple myeloma therapy Pepaxto (melphalan flufenamide).

CDER has “carefully evaluated” Pepaxto’s data and Oncopeptides’ analyses and arguments and “concluded that, for two independent reasons, the statutory standard for withdrawal is met — the confirmatory trial failed to verify clinical benefit, and the drug is no longer shown to be safe and effective under its approved conditions of use,” said the agency in its response.

“The significant safety risks … together with data failing to demonstrate benefit, decidedly show that the benefit/risk profile of melphalan flufenamide is unfavorable,” CDER’s letter continued, noting that “Apart from the risks associated with the drug itself, treatment with such a drug could delay or prevent access to alternative therapies.”

In August, Oncopeptides appealed the FDA’s formal withdrawal request, claiming that its late-stage OCEAN study showed clear clinical benefit for people with multiple myeloma, especially certain elderly patients.

Read CDER’s response letter here.

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