FDA Approves Evrysdi Tablet for Spinal Muscular Atrophy

On February 18, 2025, the U.S. Food and Drug Administration (FDA) announced the approval of a new tablet formulation of Evrysdi (risdiplam), a medication intended for individuals with spinal muscular atrophy (SMA). This tablet version is expected to hit the market in the forthcoming weeks and is designed for patients aged 2 years and older, with a body weight exceeding 44 pounds.

The decision to approve the tablet form was grounded in results from a bioequivalence study. This research indicated that the 5-mg tablet, whether swallowed whole or dissolved in filtered water, delivers a similar level of drug exposure as the previously available oral solution of Evrysdi. Despite the introduction of the tablet, the oral solution will continue to be an option for those receiving different doses or who have a preference for the liquid form.

Kenneth Hobby, the president of Cure SMA, emphasized the significant benefits of simplifying treatment administration for those affected by SMA. He highlighted that the new tablet formulation, which can be stored at room temperature, provides a practical alternative that could better align with everyday activities such as work, travel, and schooling.

The FDA's approval has been granted to Genentech, the pharmaceutical company responsible for Evrysdi. The introduction of the tablet form represents an important advancement for individuals managing SMA, offering greater flexibility and convenience in medication administration.