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Risdiplam Receives FDA Approval for Spinal Muscular Atrophy
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Key Takeaways
- FDA approved risdiplam (Evrysdi) tablet for SMA, marking it as the only non-invasive, disease-modifying treatment for patients aged 2+ and over 44 pounds.
- The 5 mg tablet can be swallowed whole or dissolved in water, offering flexibility in administration for patients.
Risdiplam is the only approved non-invasive disease-modifying SMA treatment.
Updated February 12, 2025, at 2:50PM.
The FDA approved a tablet new drug application for risdiplam (Evrysdi; Genentech), which is indicated to treat individuals with spinal muscular atrophy (SMA). This marks risdiplam as the only approved non-invasive disease-modifying SMA treatment, designated for individuals who are 2 years of age or older and weigh more than 44 Ibs.1
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The 5 mg tablet can be swallowed whole or dispersed in water and is expected to be available in the coming weeks, according to researchers from Genentech.1
“Evrysdi has robust potential to modify the SMA disease trajectory and has already been used to treat thousands of patients to date. This approval marks another significant step forward,” Levi Garraway, MD, PhD, Genentech’s chief medical officer and head of Global Product Development, said in a news release. “The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management.”1
SMA is a severe, progressive neuromuscular disease that affects the central nervous system, peripheral nervous system, and voluntary muscle movement.2 The disease is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a SMN protein deficiency. Without this protein, individuals can experience muscle weakness which could impact their physical strength and their ability to walk, eat, or breathe.1
As a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier, risdiplam is indicated to treat mutations in the chromosome 5q that causes the SMN protein deficiency. The approval of the new tablet extends from the existing oral liquid form of risdiplam, which was approved in 2020.3,4
Approval of the tablet was based on results from a bioequivalence study that demonstrated comparable exposure to risdiplam among individuals that swallowed or dispersed the 5 mg tablet into water. The results concluded that the tablet had the same effectiveness and safety as the existing oral solution. However, the study authors noted that the oral solution will still be available for patients who need different doses or prefer it.1
Risdiplam is currently being, or has been evaluated in various global multicenter trials among individuals with SMA. All of the completed studies demonstrated positive results in individuals treated with risdiplam.1
An open-label, 2-part pivotal FIREFISH (NCT02913482) clinical trial assessed the use of risdiplam among infants with type 1 SMA. The study met its primary end point and has concluded after 5 years of follow-up.1
The 2-part, double-blind, placebo-controlled pivotal SUNFISH (NCT02908685) pivotal study included individuals aged 2 to 25 years with types 2 or 3 SMA. The study also met its primary end point and concluded after 5 years follow up.1
The RAINBOWFISH (NCT03779334) open-label, single-arm, multicenter study met its primary end point. The study assessed the efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in babies from birth to 6 weeks of age with genetically diagnosed SMA that were not presenting symptoms.1
“We cannot underestimate the value that comes with simplifying treatment administration and disease management for people who are living with SMA or those caring for them,” Kenneth Hobby, president of Cure SMA, said in a news release. “This new room temperature stable formulation option offers an additional choice that may more conveniently fit into daily living activities such as working, traveling, and education.”1
