The European Commission (EC) has approved a label extension for Roche’s 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.

Earlier this year, the FDA granted approval to the NDA for the Evrydsi tablet.

“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.”

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 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.

The 5mg tablet formulation is suitable for people two years of age or older, who weigh 20kg (44 lbs) or more and are able to swallow without the use of a feeding tube. The original oral solution will remain available for those on other doses of Evrysdi and for those who may prefer the oral solution.