FDA Approves Amondys 45 for Duchenne Patients With Exon 45 Mutation

FDA Approves Amondys 45 for Duchenne Patients With Exon 45 Mutation

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FDA Approves Amondys 45 for Duchenne Patients With Exon 45 Mutation

The U.S. Food and Drug Administration (FDA) has conditionally approved Amondys 45 — formerly known as casimersen — by Sarepta Therapeutics as the first treatment for people with Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping. Accelerated, or conditional, approval is granted to a medication whose immediate availability fulfills an unmet medical need, provided early evidence of its benefits outweigh potential risks. Amondys 45’s continued use, and full approval, requires further verification of clinical benefit in the global, confirmatory Phase…

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