Roche’s Evrysdi tablet approved by European Commission as first and only for Spinal Muscular Atrophy (SMA)

• Simplified storage and administration of new tablet formulation may provide greater freedom and independence for people with SMA
• Evrysdi offers the same efficacy and safety demonstrated in available oral solution
• Evrysdi is the only non-invasive disease-modifying SMA treatment, with more than 18,000 people with SMA treated globally to date

Basel, 04 June 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Commission (EC) has approved a label extension for Evrysdi (risdiplam) to include a new, room-temperature stable tablet for people living with spinal muscular atrophy (SMA). The 5mg tablet (approx. 6.5mm), which can either be swallowed whole or dispersed in water, can be taken with or without food and does not require refrigeration, when stored at room temperature. Administered at home, Evrysdi is the only non-invasive disease modifying treatment available for people living with SMA.

“The new Evrysdi tablet with its flexible administration represents progress toward more versatile SMA disease management," said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. “With over 18,000 people treated to date, Evrysdi’s proven efficacy, safety and convenience has significantly improved the course of disease for people living with SMA.”

Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein throughout the entire central nervous system (CNS) and in peripheral tissues. Together with this innovative mode of action, the new tablet formulation offers additional portability and convenience benefits for the thousands of people living with SMA, their families and caregivers.

“We welcome the development of new treatment formulations that have the potential to further simplify disease management and care for people living with SMA,” Nicole Gusset, Chief Executive Officer, SMA Europe commented. “This is a disease requiring daily management, and it is paramount that people living with SMA, and those who care for them, are given options to optimise treatment administration.”

The approval is based on data from a bioequivalence study (NCT04718181) evaluating the 5mg tablet formulation of Evrysdi, which can either be swallowed whole or dispersed in water. Results presented at SMA Europe’s 4th Scientific International Congress in 2024 demonstrated that the tablet formulation and original oral solution provided bioequivalence to Evrysdi, meaning individuals taking the tablet can expect the same established efficacy and safety as the oral solution.