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

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.

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 boys born worldwide. Over time patients lose the ability to walk, dress, bathe and eventually breathe on their own. Most boys with DMD die when they’re young men – before the age of 30 – from respiratory or cardiac failure when lung and heart muscles give out

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