FDA Approves Roche’s Evrysdi for Spinal Muscular Atrophy

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The FDA has approved Roche’s Evrysdi (risdiplam) tablet, making it the first and only treatment for spinal muscular atrophy (SMA) available in the tablet form. According to the company, the tablet can be swallowed whole or dispersed in water, offering greater flexibility for patients. Approval was based on bioequivalence studies confirming that the tablet version of Evrysdi provides the same efficacy and safety as the existing oral solution.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,” said Levi Garraway, MD, PhD, chief medical officer, head of global product development, Genentech, in a press release. “The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management.”

Evrysdi has been evaluated in patients with SMA across multiple trials, which include:

• FIREFISH: an open-label, two-part pivotal clinical trial in infants with type 1 SMA. Participants of the study had a median age of 5.5 months during the enrollment period and included 58 infants. Only six participants were excluded from the open-label extension study.
• SUNFISH: a two-part, double-blind, placebo-controlled pivotal study in patients between two and 25 years of age with SMA types 2 or 3.
• JEWELFISH: an open-label exploratory trial aimed at evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of the drug in patients between six months and 60 years of age who received treatment prior to Evrysdi.
• RAINBOWFISH: an open-label, single-arm, multicenter study investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of Evrysdi in newborns up to six weeks of age with no present symptoms.
• MANATEE: a Phase II/III clinical study to evaluate the safety and efficacy of GYM329 in combination with Evrysdi in patients between two and 10 years of age. GYM329 targets muscle growth.
• HINALEA 1 and HINALEA 2: Phase IV clinical studies to evaluate the efficacy and safety of Evrysdi in patients under two years of age at enrollment. As part of the study, all patients were required to have previously received onasemnogene abeparvovec gene therapy.
• PUPFISH: a Phase II open-label study to investigate the pharmacokinetics and safety in patients under 20 days of age at first dose.¹

According to Medscape, SMA type 1 affects approximately one per 10,000 newborns in the United States. SMA types 2 and 3 affect one out of 24,000 newborns. SMA type 2 is the most common form of the disease, accounting for one-half of all cases, while types 1 and 3 account for one-fourth of cases. The median survival rate for SMA type 1 is seven months, with a 95% mortality rate by 18 months of age. In most cases, respiratory infections are the cause of death. SMA is more common in males, particularly with types 1 and 2. Onset is from birth to six months for type 1, between six and 18 months for type 2, after 18 months for type 3, and in the mid-30s for type 4.²

“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,” said Kenneth Hobby, president, Cure SMA, in the press 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.”

The new formulation is expected to be available in the coming weeks for patients two years of age and older who weigh more than 44 lbs.¹

References

1. FDA Approves Genentech’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA). Genentech. February 12, 2025. Accessed February 13, 2025. https://www.gene.com/media/press-releases/15052/2025-02-12/fda-approves-genentechs-evrysdi-tablet-a

2. Spinal Muscular Atrophy. Medscape. May 31, 2022. Accessed February 13, 2025. https://emedicine.medscape.com/article/1181436-overview#showall