New Tablet Formulation of Evrysdi for Spinal Muscular Atrophy Greenlighted by the FDA

A New Drug Application (NDA) for a tablet formulation of Evrysdi (risdiplam; Genentech, South San Francisco, CA) has been approved by the Food and Drug Administration (FDA) for the treatment of participants aged ≥2 years with spinal muscular atrophy (SMA) who weigh ≥20 kg. Evrysdi was first approved in 2020 as an oral solution for children aged ≥2 months, and its indication was expanded in 2022 to include children aged <2 months. The new 5 mg tablet formulation can be swallowed whole or dispersed in nonchlorinated drinking water, and according to a statement from Genentech, will be available in the coming weeks in the United States.

The approval is based on data reported from a bioequivalence study demonstrating that treatment exposure for Evrysdi tablets, either swallowed whole or dispersed in water, is comparable to the oral solution formulation. The findings of this study suggest that patients with SMA who are treated with Evrysdi tablets can expect the same efficacy and safety outcomes as they would with the original oral solution. Additionally, the new tablet formulation is stable at room temperatures.

“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.”