“Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date. This approval marks another significant step forward,” said Levi Garraway, M.D., Ph.D., Genentech’s Chief Medical Officer and Head of Global Product Development. “The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management.”
Today, Genentech, a member of the Roche Group, 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).
The Company also shared an SMA Community Letter that highlights our community's request for greater indepedence as a driving factor in the pursuit of Evrysdi tablet.
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.
"We cannot underestimate the value that comes with simplifying treatment administration and disease management for people who are living with SMA or those caring for them,” said Kenneth Hobby, President of Cure SMA. "This new room temperature stable formulation option offers an additional choice that may more conveniently fit into daily living activities such as working, traveling, and education."
The new tablet, expected to be available in the coming weeks, is suitable for people two years of age or older who weigh more than 20 kgs (44 lbs).
Next Steps
On Tuesday, March 11, at 12:00pm CT, Cure SMA will co-host a webinar with Genentech on the FDA approval of Evrysdi tablet. Click here to register and submit your questions.
In August 2024, Cure SMA hosted a webinar for the community that reviewed the important steps that will follow positive results from clinical trials that could lead to regulatory filing and potentially next treatment approvals. Click here to see a recording.
Cure SMA will continue to share updates as the Evrysdi tablet becomes available to the community.