FDA Approves Genentech’s Treatment for Spinal Muscular Atrophy (SMA) in Adults and Children 2 Months and Older
August 7th, 2020
On August 7, 2020, the FDA approved Genentech’s Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. Evrysdi showed clinically-meaningful improvements in motor function across two clinical trials in people with varying ages and levels of disease severity, including Types 1, 2, and 3 SMA. A liquid medicine, Evrysdi is administered daily at home by mouth or feeding tube.
News in brief
- In two clinical trials, Evrysdi improved motor function in people living with SMA over a broad spectrum of ages and levels of disease severity, including Types 1, 2, and 3 SMA
- Evrysdi is the first and only medicine for SMA that can be taken at home
- SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality.
Supporting Information
“Given the majority of people with SMA in the U.S. remain untreated, we believe Evrysdi, with its favorable clinical profile and oral administration, may offer meaningful benefits for many living with this rare neurological disease.”