On Feb. 12, 2025, Genentech announced that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for an Evrysdi^® (risdiplam) tablet for people living with spinal muscular atrophy (SMA).

Evrysdi is the only non-invasive disease-modifying treatment for SMA. The 5 mg Evrysdi tablet can either be swallowed whole or dispersed in water. 

The approval of the Evrysdi tablet was based on the results of a bioequivalence study, which demonstrated that the 5 mg tablet, whether swallowed whole or dispersed in non-chlorinated drinking water (e.g., filtered water), and original oral solution provide comparable exposure to risdiplam. This means patients who take the tablet can expect the same established efficacy and safety as the Evrysdi oral solution. The Evrysdi oral solution will remain available for those on other doses of Evrysdi and for those who may prefer the oral solution.

Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the central nervous system (CNS) and peripheral tissues, as demonstrated in animal models. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and core functions.