FDA withdraws approval of Oncopeptides’ Pepaxto


The US Food and Drug Administration (FDA) has announced its final decision to withdraw approval of Pepaxto (melphalan flufenamide), despite an appeal by manufacturer Oncopeptides.

The drug was originally approved for use in combination with dexamethasone to treat some patients with multiple myeloma.

Approval was withdrawn as the confirmatory study conducted as a condition of accelerated approval did not confirm Pepaxto’s clinical benefit, and Pepaxto has not been proved to be safe or effective under its conditions of use.

This is the first time the FDA has used the amended procedures for withdrawal of accelerated approval that were enacted in 2023, as part of the Food and Drug Omnibus Report Act of 2022 (FDORA).

Pepaxto associated with increased risk of death

The FDA is alerting patients and health care professionals that a clinical trial (OCEAN, Study OP-103) evaluating Pepaxto with dexamethasone showed an increased risk of death.

The trial compared Pepaxto with low-dose dexamethasone 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.

In February 2021, the FDA approved Pepaxto under Accelerated Approval for use in combination with dexamethasone to treat adult patients with relapsed or refractory multiple myeloma who have 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.

Oncopeptides was required to conduct the OCEAN trial as a post-approval requirement under the accelerated approval programme.

The FDA has now suspended enrolment in all ongoing Pepaxto clinical trials. Patients receiving clinical benefit from Pepaxto may continue treatment in the OCEAN trial provided they are informed of the risks and sign a revised written informed consent.

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