New York, Feb. 12, 2025 (GLOBE NEWSWIRE) — The Muscular Dystrophy Association (MDA) today celebrates the decision by the US Food and Drug Administration (FDA) to grant approval of a risdiplam (Evrysdi) in tablet form, marking a significant advancement in the treatment of spinal muscular atrophy (SMA), the leading genetic cause of infant death. Read Genentech’s press release here.
This new formulation provides an additional treatment option for pediatric and adult patients, making the therapy more accessible and convenient in tablet form. Evrysdi, marketed by Genentech, a Roche company, is a disease-modifying therapy designed to target the genetic root cause of SMA by increasing the production of the missing functional survival motor neuron (SMN) protein.
The approval of this new tablet builds on previous successes in SMA treatment, joining the existing oral liquid version of risdiplam (Evrysdi), as well as Biogen’s nusinersen (Spinraza) and Novartis Gene Therapies’ onasemnogene abeparvovec-xioi (Zolgensma). The new tablet formulation expands treatment options, offering patients and families easier access to treatment and increased flexibility in managing SMA.
“Muscular Dystrophy Association celebrates continued advancements in SMA treatment, ensuring patients have access to effective and convenient therapies,” said Sharon Hesterlee, PhD, EVP, Chief Research Officer, MDA. “The approval of the tablet formulation of Evrysdi marks another major milestone for the SMA community, providing an easier administration method that enhances quality of life for people living with this disease.”
Evrysdi’s development benefits from years of research into SMA biology funded by MDA. MDA has committed more than $50 million to SMA-related projects and over $1 billion to neuromuscular disease research as a whole. The pace of drug development continues to accelerate, offering new hope for SMA and other neuromuscular diseases.
Study Supported Approval of Evrysdi Tablet Formulation
According to Genentech’s press release, the approval of the Evrysdi tablet was based on the results of a bioequivalence study, which demonstrated that the 5 mg tablet, whether swallowed whole or dispersed in non-chlorinated drinking water (e.g., filtered water), and original oral solution provide comparable exposure to risdiplam. This means patients who take the tablet can expect the same established efficacy and safety as the Evrysdi oral solution. The Evrysdi oral solution will remain available for those on other doses of Evrysdi and for those who may prefer the oral solution.
The study was originally presented at the 2024 MDA Clinical & Scientific Conference, view the abstract here.
About Spinal Muscular Atrophy (SMA)
SMA is caused by a mutated or missing survival motor neuron 1 (SMN1) gene, leading to insufficient production of SMN protein and resulting in motor neuron loss, muscle weakness, and paralysis. SMA is classified into four types based on disease onset and severity, with type 1 being the most severe.
About Evrysdi
Evrysdi is an SMN2-splicing modifier designed to increase functional SMN protein production. With the introduction of a new tablet formulation, Evrysdi now offers an alternative administration method for patients, enhancing treatment adherence and accessibility.
MDA Resource Center
MDA Resource Center is available to provide one-on-one support via phone or email for individuals and families looking for an overview of disease information, resources, activities and access to care in the United States through the MDA Care Center Network.
Media inquiries contact press@mdausa.org.
About Muscular Dystrophy Association
Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and any of over 300 other neuromuscular conditions. For 75 years, MDA has led the way in accelerating research, advancing care, and advocating support and inclusion of families living with neuromuscular disease. MDA’s mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, X, Threads, TikTok, LinkedIn, and YouTube.
About Muscular Dystrophy Association’s 75th Anniversary
In 2025, the Muscular Dystrophy Association proudly marks 75 years legacy, impact and momentum in the fight against neuromuscular diseases. Since our founding, MDA has been at the forefront of research breakthroughs, providing access to comprehensive care, and championing the rights of people living with muscular dystrophy, ALS, and over 300 other neuromuscular diseases. This milestone has been made possible by generations of dedicated support from people living with neuromuscular disease, their families, researchers, clinicians, volunteers, and donors—who boldly drive our mission forward. As we look ahead, we remain committed to honoring this legacy, building on the impact we’ve made together, and continuing our momentum toward transformative progress for people living with neuromuscular disorders. Learn more at MDA75.org.
- MDA – New FDA Treatment Approved for SMA
