Roche’s Evrysdi Tablet Gains European Commission Approval for Spinal Muscular Atrophy
Wednesday, June 04, 2025
Roche has received approval from the European Commission for a new 5mg tablet formulation of Evrysdi® (risdiplam), the first and only non-invasive, disease-modifying treatment for spinal muscular atrophy (SMA).
Spinal muscular atrophy is a rare and progressive neuromuscular disorder caused by a mutation in the SMN1 gene, leading to a deficiency in SMN protein. This deficiency disrupts the functioning of nerves that control muscle movement, potentially affecting the ability to walk, eat, or breathe. SMA affects roughly one in 10,000 newborns and remains a leading genetic cause of infant death.
The tablet is stable at room temperature, can be swallowed whole or mixed with water, and does not require refrigeration. It offers greater flexibility and convenience for people living with SMA, supporting at-home administration and easing daily treatment routines.
Evrysdi remains the only non-invasive, disease-modifying treatment available for SMA and has already been used to treat over 18,000 people globally. This new formulation further enhances ease of administration, particularly for those aged two years and above who weigh at least 20kg and can swallow tablets without a feeding tube.
The tablet formulation demonstrated bioequivalence with the original oral solution, as confirmed by data from a clinical study (NCT04718181) presented at SMA Europe’s Scientific International Congress in 2024. This means it offers the same safety and effectiveness as the liquid version. The original oral solution remains available for those on different doses or who prefer it.
Evrysdi works by boosting and maintaining the production of SMN protein in both the central nervous system and peripheral tissues. This protein is essential for motor neuron health and overall muscle function.
Roche continues to lead the development of Evrysdi in collaboration with the SMA Foundation and PTC Therapeutics.
The treatment has received numerous regulatory designations, including PRIME status from the European Medicines Agency and Orphan Drug Designation from the U.S. FDA.
Source: roche.com
