Duchenne muscular dystrophy
The FDA will close out a hectic month of March with a flurry of target action dates, including ones for lymphoma and CKD anemia treatments.
The recently launched company shared two posters at the Muscular Dystrophy Association’s annual conference this week ahead of a March 21 PDUFA date.
Sarepta Therapeutics on Wednesday called the launch of the gene therapy for Duchenne muscular dystrophy “exceptional” but the company does not expect to see significant growth in the first half of 2024 due to its currently limited patient pool.
The regulator has granted a priority review of the efficacy supplement for Sarepta Therapeutics’ gene therapy Elevidys with a target decision date of June 21, 2024.
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