FDA Approves Evrysdi Tablet from Roche for Spinal Muscular Atrophy

FDA has approved a new drug application for Roche’s Evrysdi (risdiplam) tablet for treating people living with spinal muscular atrophy (SMA), which makes the drug the first and only tablet for treating SMA, Roche announced on Feb. 12, 2025. Risdiplam, offered in a 5 mg tablet that can be swallowed whole or dispersed in water, is also the only non-invasive disease-modifying treatment in this indication, according to Roche in a company press release (1).

FDA’s approval was based on the results of a bioequivalence study. This study demonstrated that the 5 mg tablet—swallowed whole or dispersed in non-chlorinated drinking water (e.g., filtered water)—provided comparable exposure to risdiplam compared with the original risdiplam oral solution. Thus, patients who take the tablet can expect the same efficacy and safety that is established with the oral solution. The oral solution will remain available for those taking other doses of the drug and for those who may prefer the oral solution.

"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 and head of Global Product Development, Roche, in the press release (1). "The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management."

The new 5 mg tablet is expected to be made available in the coming weeks following its approval. It is suitable for use in people two years of age or older who weigh more than 20 kg (44 lbs). Roche is responsible for the clinical development of risdiplam (under the brand name Evrysdi) as part of a collaboration with the SMA Foundation and PTC Therapeutics.

"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 of Cure SMA. "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."

Risdiplam (under the brand name Evrysdi) is approved in more than 100 countries. An important benefit of the tablet formation is that it can be stored at room temperature, compared to the oral formulation. This benefit may provide greater freedom and independence for people dealing with SMA.

Risdiplam is a survival motor neuron 2 (SMN2) pre-messenger RNA splicing modifier. It is a small-molecule drug (2) designed to treat SMA resulting from mutations in chromosome 5q. Such mutations can lead to survival of motor neuron (SMN) protein deficiency. The drug is administered daily either at home or in transit. In oral solution form, it is administered by mouth or via a feeding tube; with the new tablet form it is administered by mouth only, either swallowed whole or dispersed in water.

The European Medicines Agency granted risdiplam priority medicines (PRIME) designation in 2018, and FDA granted it orphan drug designation in 2017. The drug is currently being, or has been, evaluated in a number of global multicenter trials in people with SMA (1).

References

1. Roche. FDA Approves Roche’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA). Press Release. Feb. 12, 2025.
2. Ratni, H.; Scalco, R. S.; Stephan, A.H. Risdiplam, the First Approved Small Molecule Splicing Modifier Drug as a Blueprint for Future Transformative Medicines. ACS Med. Chem. Lett. 2021, 12 (6), 874–877. DOI: 10.1021/acsmedchemlett.0c00659