The newly approved Evrysdi tablet formulation demonstrated bioequivalence to the oral solution and provides comparable systemic exposure, efficacy, and safety in an alternative administration option for patients with spinal muscular atrophy.

Roche announced that the U.S. Food and Drug Administration (FDA) has approved a tablet formulation of Evrysdi® (risdiplam), a survival motor neuron 2 pre-mRNA splicing modifier, for the treatment of spinal muscular atrophy (SMA). The 5 mg tablet formulation provides the same efficacy and safety profile as the currently available oral solution and is approved in patients aged 2 years and older who weigh at least 20 kg (44 lbs). 

The approval was based on a bioequivalence study conducted by researchers at Roche in collaboration with the SMA Foundation and PTC Therapeutics. They assessed systemic plasma exposure of risdiplam in patients taking the tablet formulation vs those receiving the oral solution. The tablet demonstrated comparable exposure to the oral solution in the bioequivalence study.

The tablet can be swallowed whole or dispersed in nonchlorinated water to ensure flexibility in administration. The researchers also confirmed that adverse event profiles were consistent between both formulations. No new safety concerns were identified.

The tablet formulation offers a significant advantage to the oral solution by eliminating the need for refrigeration and enabling storage at room temperature. “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,” said Kenneth Hobby, President of Cure SMA, in a press release from Roche.

Evrysdi is currently approved in over 100 countries, and more than 16,000 patients have been treated worldwide. Roche continues to investigate combination therapies, such as GYM329 (RG6237)—an anti-myostatin molecule targeting muscle growth—to further enhance SMA treatment outcomes. The new tablet formulation is expected to be available in the coming weeks. Updated prescribing information now reflects guidance on tablet administration and storage.