• 05 Jun 2025
• Admin
• News Article

European Commission approves Roche’s Evrysdi tablet to treat spinal muscular atrophy

Roche has announced that the European Commission (EC) has approved a label extension for Evrysdi (risdiplam), enabling the use of a new room-temperature stable tablet for the treatment of spinal muscular atrophy (SMA).

The newly approved 5mg tablet, measuring approximately 6.5mm, offers a convenient administration option that can be swallowed whole or dispersed in water, taken with or without food, and stored without refrigeration.

Evrysdi remains the only non-invasive, at-home, disease-modifying therapy available for people with SMA.

Enhanced Flexibility and Convenience in SMA Management

Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development at Roche, noted that the flexible administration of the new tablet formulation marks a significant step forward in SMA disease management.

With over 18,000 individuals treated worldwide, Evrysdi has demonstrated proven efficacy, safety, and patient convenience in modifying the course of SMA.

Mechanism of Action and Patient-Centric Advancements

Evrysdi functions by increasing and maintaining the production of SMN protein across the central nervous system and peripheral tissues. The SMN protein is vital for the health and functionality of motor neurons.

The new tablet offers an added layer of portability and convenience, particularly beneficial for patients, families, and caregivers navigating the daily challenges of SMA.

Nicole Gusset, CEO of SMA Europe, emphasized that simplifying treatment administration is essential to improving the quality of care and life for individuals living with SMA. With this new formulation, patients and caregivers have additional choices to optimize treatment.

Clinical Data and Bioequivalence Study

The European Commission’s decision was based on findings from a bioequivalence study (NCT04718181), which confirmed that the new 5mg tablet formulation delivers the same efficacy and safety as the original oral solution. These results were presented at the 4th Scientific International Congress of SMA Europe in 2024.

The tablet formulation is indicated for individuals aged two years and older, weighing at least 20kg (44 lbs), and capable of swallowing without the use of a feeding tube. Roche will continue offering the original oral solution for those who require or prefer this method.

Collaborative Development and Mode of Action

Evrysdi is being developed by Roche in collaboration with the SMA Foundation and PTC Therapeutics. It is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by 5q-related mutations leading to SMN protein deficiency. The medication works by supporting the sustained production of the SMN protein, which is essential for muscle function and neuronal health.

It is administered once daily at home in either a liquid or tablet form, providing a consistent and manageable treatment regimen.

Regulatory Recognition and Global Reach

Evrysdi has received numerous regulatory designations:

• PRIME designation from the European Medicines Agency (EMA) in 2018
• Orphan Drug Designation from the US Food and Drug Administration (FDA) in 2017
• Drug Discovery of the Year award from the British Pharmacological Society in 2021
• Society for Medicines Research Award for Drug Discovery in 2021

Currently, Evrysdi is approved in more than 100 countries, with more than 18,000 patients treated globally.

Ongoing and Completed Clinical Trials for Evrysdi

Evrysdi has been or is currently being evaluated in a range of global multicenter trials across all ages and SMA types:

FIREFISH (NCT02913482)

• A two-part, open-label pivotal trial in infants with Type 1 SMA
• Included 58 infants; 52 continued into the extension study
• Primary endpoint met; study concluded after 5 years

SUNFISH (NCT02908685)

• A double-blind, placebo-controlled pivotal study for Types 2 and 3 SMA in patients aged 2–25 years
• Met its primary endpoint and concluded after 5 years

JEWELFISH (NCT03032172)

• An open-label exploratory study evaluating safety and pharmacokinetics in SMA patients aged 6 months to 60 years who had prior SMA therapies
• Recruitment completed (n=174)

RAINBOWFISH (NCT03779334)

• Single-arm, open-label trial for presymptomatic infants (birth to 6 weeks) with genetically confirmed SMA
• Enrolled 26 babies; study met its primary endpoint

MANATEE (NCT05115110)

• Phase II/III study combining Evrysdi with GYM329, an anti-myostatin molecule, in SMA patients aged 2–10 years
• GYM329 received Orphan Drug Designation in December 2021
• Study is actively recruiting

HINALEA 1 & 2 (NCT05861986, NCT05861999)

• Phase IV studies assessing safety and effectiveness in infants who received gene therapy (onasemnogene abeparvovec) either pre- or post-symptomatically
• Currently recruiting

PUPFISH (NCT05808764)

• Phase II, open-label study in newborns under 20 days old at first dose
• Focuses on pharmacokinetics and safety
• Currently recruiting

Overview of Spinal Muscular Atrophy (SMA)

SMA is a rare, progressive, and often fatal neuromuscular disorder affecting approximately one in 10,000 babies. It is the leading genetic cause of infant mortality.

SMA is caused by mutations in the SMN1 gene, resulting in a deficiency of SMN protein, which is essential for the function of motor neurons.

Without adequate SMN protein, motor neurons deteriorate over time, leading to muscle weakness and loss of critical functions such as walking, eating, or breathing. The disease requires ongoing management and treatment to preserve quality of life.

Roche's Commitment to Neuroscience

Neuroscience remains a core focus of research and development at Roche. The company is dedicated to advancing treatments for chronic and complex neurological conditions. Roche is currently investigating more than a dozen therapies across various disorders, including:

• Multiple sclerosis
• Spinal muscular atrophy
• Neuromyelitis optica spectrum disorder
• Alzheimer’s disease
• Huntington’s disease
• Parkinson’s disease
• Duchenne muscular dystrophy

Through internal research and external partnerships, Roche continues to push the boundaries of science to address some of the most pressing challenges in neurology.