> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# Genentech’s Spinal Muscular Atrophy Drug Approved
- URL: https://www.fdaweb.com/genentechs-spinal-muscular-atrophy-drug-approved/
- Published: 2020-08-07T12:00:00.000Z
- Updated: 2026-09-14T16:24:17.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5147385

FDA has approved Genentech’s Evrysdi (risdiplam) for treating spinal muscular atrophy (SMA) in adults and children two months of age and older. SMA is a rare and often fatal genetic disease affecting muscle strength and movement. The hereditary disease causes weakness and muscle wasting because patients lose lower motor neurons that control movement.

Approval was based on data from two clinical studies. In one trial, data showed that after 12 months of treatment “41% of patients were able to sit independently for more than five seconds, a meaningful difference from the natural progression of the disease because almost all untreated infants with infantile-onset SMA cannot sit independently,” an [agency release](http://s2027422842.t.en25.com/e/es?s=2027422842&e=361030&elqTrackId=376c7bc788024cd5a73d955f2e3dcbdc&elq=e1431265ddb6475dbdd76eeeaadff2f0&elqaid=13711&elqat=1&ref=fdaweb.com) says. “After 23 or more months of treatment, 81% of patients were alive without permanent ventilation, which is a noticeable improvement from typical disease progression without treatment.” The second study, a randomized, placebo-controlled study, saw patients on Evrysdi having an average 1.36 increase in their motor function score after one year, compared to a 0.19 decrease in patients on placebo, FDA says.