European Commission Approves New Evrysdi Tablet, Offering Enhanced Flexibility for SMA Treatment

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European Commission Approves New Evrysdi Tablet, Offering Enhanced Flexibility for SMA Treatment

(IN BRIEF) Roche announced the European Commission’s approval of a label extension for Evrysdi® (risdiplam) to include a new 5mg tablet, offering a room-temperature stable alternative to the original oral solution for individuals with spinal muscular atrophy (SMA). The tablet, which can be taken whole or dissolved in water, provides the same proven efficacy and safety as the oral solution. This new formulation is suitable for individuals aged two years or older, making SMA treatment more convenient and accessible. Over 18,000 people worldwide have been treated with Evrysdi, which continues to be the only non-invasive, disease-modifying treatment for SMA. The approval reflects Roche’s commitment to improving SMA disease management and providing more flexible treatment options for patients.

(PRESS RELEASE) BASEL, 4-Jun-2025 — /EuropaWire/ — Roche (SIX: RO, ROG; OTCQX: RHHBY), a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives, has announced that the European Commission (EC) has approved a label extension for Evrysdi® (risdiplam), introducing a new, room-temperature stable tablet for individuals living with spinal muscular atrophy (SMA). The 5mg tablet, which can be taken either whole or dissolved in water, does not require refrigeration and can be consumed with or without food. It is a non-invasive, disease-modifying treatment, marking a significant milestone for SMA management.

Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development at Roche, commented, “The introduction of the Evrysdi tablet offers a flexible and convenient option for SMA patients, making disease management more accessible. With over 18,000 individuals treated globally, Evrysdi continues to demonstrate proven efficacy, safety, and convenience, improving the lives of people with SMA.”

The tablet’s bioequivalence to the original oral solution was confirmed through a bioequivalence study (NCT04718181), which showed that both the tablet and the oral solution provide the same established efficacy and safety. This new formulation is suitable for individuals aged two years and older, weighing at least 20kg (44 lbs), who can swallow the tablet without needing a feeding tube. The original liquid formulation of Evrysdi will remain available for individuals on other doses or those who prefer the oral solution.

Evrysdi works by enhancing the production of SMN protein, crucial for maintaining motor neurons and enabling muscle function. This tablet offers improved portability and ease of use, benefiting patients and caregivers alike. Nicole Gusset, CEO of SMA Europe, expressed, “This new formulation of Evrysdi is a step forward in simplifying SMA disease management, offering more flexibility and convenience for patients and their families.”

The approval of the tablet is based on positive clinical data, and it is expected to further streamline SMA treatment, helping patients live more independently. Roche continues its collaboration with the SMA Foundation and PTC Therapeutics to advance the clinical development of Evrysdi.

About SMA
SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to muscle weakness over time. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.

About Roche in Neuroscience
Neuroscience is a major focus of research and development at Roche. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases.

Roche is investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today.

About Roche
Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice.

For over 125 years, sustainability has been an integral part of Roche’s business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045.

Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan.

For more information, please visit www.roche.com.

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