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

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 a mutation amenable to exon 45 skipping