Evrysdi is the first, and so far only, noninvasive disease-modifying treatment for spinal muscular atrophy.
The FDA on Wednesday approved the industry’s first-ever tablet treatment for spinal muscular atrophy—Roche’s SMN2 splicing modifier Evrysdi—offering patients a more convenient and flexible dosing option.
The approval, granted to Roche subsidiary Genentech, makes Evrysdi the only noninvasive disease-modifying therapy for spinal muscular atrophy (SMA), according to the company’s news release. The tablet, which can be taken whole orally or dissolved in water, will be available in the coming weeks, the company said, and can be given to patients aged 2 years and up who weigh over 20 kgs (44 lbs).
Genentech chief medical officer Levi Garraway called the approval a “significant step forward” for SMA patients. “Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date,” Garraway said, adding that the tablet formulation “combines established efficacy with convenience.”
Everysdi’s active ingredient is risdiplam, a small-molecule drug that promotes the production of full-length and functional SMN protein in the brain. SMN—also known as survival of motor neuron—is involved in repairing DNA, managing cellular stress and preserving overall neuromuscular function. In SMA, genes encoding for SMN are typically mutated, leading to dysfunctional protein expression.
The drug was first approved in 2020, but as an oral solution that must be constituted by a healthcare provider before being dispensed to patients, according to its label. The drug is administered once daily after a meal through an oral syringe.
Data from a bioequivalence study supported the approval of the pill form. Results showed that a 5-mg tablet could elicit comparable exposure to risdiplam, suggesting that patients taking the tablet will see “the same established efficacy and safety” as the oral solution of Evrysdi, according to Wednesday’s release.
The pill approval for Evrysdi bucks Roche’s recent losing streak in the neurodegenerative space. In December 2024, the pharma announced that its Prothena-partnered Parkinson’s disease candidate prasinezumab failed its Phase IIb trial. The pharma pointed to “potential clinical efficacy” on the primary endpoint—time to confirmed motor progression—though the effect was not statistically significant.
In October 2024, Roche let go of an Alzheimer’s disease prospect, sending rights to bepranemab back to UCB.
Elsewhere in the SMA space, Biogen last month announced that the FDA had accepted a supplemental filing for Spinraza that proposes a higher dose of the Ionis-partnered asset for SMA. In October, Scholar Rock aced Phase III testing for its myostatin blocker apitegromab, which elicited significant improvements in motor function in kids with SMA.
