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DMD Pipeline: After Sarepta’s First-Ever Approval, Are Combinations Next?

 
• By Mandy Jackson

Several companies have DMD drug candidates waiting in the wings to follow Sarepta’s Exondys 51 into the commercial market after FDA approval of the exon-skipping therapy, which could be the backbone for future combination treatment regimens.

DNA

Sarepta Therapeutics Inc.’s recent first-ever US FDA approval for a drug to treat Duchenne muscular dystrophy (DMD) is lending confidence for a wave of potential therapies and combination regimens to treat the rare disease – a small, but potentially lucrative market.

DMD is a devastating genetic disorder characterized by progressive muscle degeneration that affects one out of every 3,500 to 5,000...

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