Roche has announced that the European Commission (EC) has approved a label extension for Evrysdi® (risdiplam) to include a new 5mg tablet formulation designed for easier administration and improved convenience for people living with spinal muscular atrophy (SMA).
This new tablet, approximately 6.5mm in size, can be swallowed whole or dispersed in water, taken with or without food, and stored at room temperature, eliminating the need for refrigeration. It is suitable for individuals aged two years and older, weighing at least 20kg (44 lbs), and able to swallow without the aid of a feeding tube. The original oral solution will continue to be available for those on other dosages or who prefer the liquid form.
Evrysdi is the only non-invasive, at-home disease-modifying treatment for SMA. It works by increasing and maintaining SMN protein production throughout the central nervous system and peripheral tissues, which is essential for motor neuron survival.
The European approval is based on data from a bioequivalence study (NCT04718181), which confirmed that the new tablet offers comparable efficacy and safety to the original oral solution. Results were presented at the SMA Europe 4th Scientific International Congress in 2024.
Nicole Gusset, CEO of SMA Europe, welcomed the update: “SMA requires daily management, and it is vital that treatment options reflect the real-world needs of patients and caregivers. This new tablet offers greater flexibility in how treatment is administered.”
Evrysdi is developed by Roche in collaboration with the SMA Foundation and PTC Therapeutics, reinforcing the company’s commitment to enhancing the quality of life for people with SMA.