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Oncopeptides’ Pepaxto Needs New Study To Identify Population That Will Benefit, FDA Panel Says

 
• By Sue Sutter

Advisory committee votes 14-2 that melflufen’s benefit-risk profile is unfavorable in the current accelerated approval indication for fifth-line treatment of multiple myeloma; panel rejects sponsor’s attempt to rely on post hoc analyses to 'carve out' a subpopulation that, it asserts, is responsible for negative overall survival trend in OCEAN confirmatory trial.

Dim room
Pepaxto's near-term future in the US appears dim following a strongly negative advisory committee vote. • Source: Shutterstock

Oncopeptides AB should conduct a prospective, randomized trial to identify an appropriate patient population for Pepaxto (melphalan flufenamide, or melflufen) because the multiple myeloma drug’s benefit-risk profile in its current indication is not favorable, a US Food and Drug Administration panel said on 22 September.

In a 14-2 vote finding an unfavorable benefit-risk profile, the Oncologic Drugs Advisory Committee rejected Oncopeptides' bid to add a limitation of use to the current indication in an effort to “carve out” those patients who, its post hoc analyses suggest, would not benefit from therapy – specifically, those who

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