The U.S. Food and Drug Administration (FDA) has granted approval to Roche’s New Drug Application (NDA) for Evrysdi (risdiplam), a tablet specifically designed for individuals living with spinal muscular atrophy (SMA). Evrysdi stands as the sole non-invasive treatment option that modifies the disease’s progression. The 5mg Evrysdi tablet can be swallowed whole or dispersed in water.

“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., Chief Medical Officer and Head of Global Product Development, Roche.

The approval of the Evrysdi tablet was based on the results of a bioequivalence study, which demonstrated that the 5mg tablet, whether swallowed whole or dispersed in non-chlorinated drinking 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.

As part of the label extension, the Evrysdi prescribing information has been updated to include guidance on tablet administration and storage.

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).