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Sarepta’s Next-Generation Drugs Draw Attention Amid ‘Incremental’ Approval

Amondys 45 Gets DMD Nod From FDA

 
• By Alaric DeArment

The RNA drug’s accelerated approval brings Sarepta’s exon-skipping market share to nearly 30%.

MUSCULAR DYSTROPHY words written on label tag with medicine,syringe,keyboard and stethoscope
Sarepta's Amondys 45 won accelerated FDA approval for DMD patients ameable to exon 45 skipping • Source: Shutterstock

Sarepta Therapeutics, Inc. gained another inch with the US Food and Drug Administration approval of its third drug for Duchenne muscular dystrophy, but the mile ahead may come in the form of next-generation therapies for DMD. For some of them, data could emerge even before confirmatory data arrives for the company’s approved first-generation drugs.

The FDA granted accelerated approval to the Cambridge, MA-based biotech’s Amondys 45 (casimersen) on 25 February, for DMD patients with...

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