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The Food and Drug Administration on Friday approved the first oral therapy for spinal muscular atrophy, a rare and often devastating disease, adding a new option for patients who now rely on costly injected treatments.

The drug, from partners Genentech and PTC Therapeutics, is approved for adults and children with SMA, regardless of the severity of their disease. The once-a-day treatment will compete with a one-time gene therapy from Novartis and a drug from Biogen that is injected into the spine every four months.


Genentech and PTC’s treatment, branded Evrysdi, is dosed based upon weight, meaning its cost will vary patient by patient. In a statement, Genentech said Evrysdi’s maximum price would be $340,000 per year. For patients who weigh less than 44 pounds, the cost would be less. Treatment for patients under 2 years old would generally cost less than $100,000 a year, according to the company.

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