New entrant in spinal muscular atrophy market, with FDA approval for Evrysdi

The US Food and Drug Administration today approved Evrysdi (risdiplam) to treat patients two months of age and older with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement. This is the first oral drug approved to treat this disease.

Evrysdi the first at-home, orally administered treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older, said PTC Therapeutics (Nasdaq: PTCT), which developed the drug as part of a collaboration with Swiss pharma giant Roche (ROG: SIX) and the SMA Foundation, and saw its shares gain 1.7% to $47.83 on Friday. The FDA granted this approval of Evrysdi to Roche subsidiary Genentech.

“Evrysdi is the first drug for this disease that can be taken orally, providing an important treatment option for patients with SMA, following the approval of the first treatment for this devastating disease less than four years ago,” said Dr Billy director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research.