PBMs Weigh Coverage of New Muscular Dystrophy, Hemophilia A Gene Therapies

The FDA last month approved two new gene therapies for Duchenne Muscular Dystrophy (DMD) and hemophilia A, and major PBMs tell AIS Health, a division of MMIT, that they have not yet decided how to cover the new treatments. If current trends are any indication, health plans will impose strict utilization management requirements and attempt to negotiate outcomes-based reimbursement pacts with the treatments’ manufacturers.

The FDA on June 22 granted accelerated approval to Sarepta Therapeutics, Inc.’s Elevidys (delandistrogene moxeparvovec-rokl), the DMD treatment. It will have a list price of $3.2 million. BioMarin Pharmaceutical Inc.’s Roctavian (valoctocogene roxaparvovec), for hemophilia A, gained full FDA approval on June 29, and will have a list price of $2.9 million. Both therapies are administered as a one-time dose. Roctavian also typically requires an indefinite period of simultaneous treatment with corticosteroids to reduce patients’ immune-system response to the gene therapy.

© 2024 MMIT
Peter Johnson

Peter Johnson

Peter has been a reporter for nearly a decade. Before joining AIS Health, Peter covered a wide variety of topics in his hometown of Seattle, where he continues to live. Peter’s work has appeared in publications including The Atlantic and The Stranger. Peter attended Colby College.

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