Evrysdi is a non-invasive, disease-modifying option that can be swallowed whole or dispersed in water for patients with spinal muscular atrophy.
Image Credit: Adobe Stock Images/john
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:
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
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