FDA Approves First Oral Treatment for Spinal Muscular Atrophy
The U.S. Food and Drug Administration approved Evrysdi from PTC Therapeutics (NASDAQ: PTCT) and Roche (OTC: RHHBY), making it the third treatment approved for patients with spinal muscular atrophy (SMA) and the first option that patients can take at home. Evrysidi, formerly known as risdiplam, is an easy-to-swallow treatment that allows SMA patients to produce functional copies of the survival motor neuron protein they lack. Roche and PTC Therapeutics set the list price of their new rare disease drug at a relatively modest $340,000 per year to compete with already approved treatments from Biogen (NASDAQ: BIIB) and Novartis (NYSE: NVS).
