FDA Approves Risdiplam as Table for Spinal Muscular Atrophy

The FDA approved risdiplam (Evrysdi) in a tablet formulation for the treatment of spinal muscular atrophy (SMA). This marks the first approval for the only non-invasive disease-modifying treatment for SMA.¹

This marks the first approval for the only non-invasive disease-modifying treatment for SMA. | Image Credit: Aquir | stock.adobe.com

"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," Levi Garraway, MD, PD, chief medical officer and head of global product development at Roche, said in a news release. "The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management."¹

The approval of the drug was based on results from a bioequivalence study, showing that the 5 mg tablet, either swallowed whole or dispersed in non-chlorinated drinking water, and the original oral solution demonstrated comparable exposure to the drug. The results demonstrated that the tablet is expected to have the same established efficacy and safety as the oral solution, which will remain available for those on other doses and who may prefer oral solutions.¹

The study (NCT04718181) was a randomized, single oral dose, crossover study that included 3 parts. Investigators aimed to determine the relative bioavailability and bioequivalence of 2 different formulations of the risdiplam tablet compared with the current oral solution for healthy patients. Investigators also studied the effect of food on the tablets and the effect of omeprazole (Prilosec, Losec) on the tablets.²

In part 1, the study was exploratory for the tablet formulation compared with the oral solution. The effect on food bioavailable, comparing fed and fasted states, was also studied. Investigators also studied the effects that omeprazole, an antacid, had on the medication. In part 2, investigators further evaluated the bioequivalence, with each patient group receiving randomized treatment in a 4-way crossover. The treatment options included the risdiplam 5 mg tablet in both fed and fasted states and a single dose of the selected dispersible tablet in both fed and fasted states. Part 3 assessed the bioequivalence in the same manner as part 2.²

The new table will be available within the coming weeks and is indicated for patients 2 years and older who weigh more than 20 kgs.¹

Investigators found that the formulation showed bioequivalence to the current available oral solution, including area under the concentration-time curve and maximum concentration. Further, there was no impact of food on the exposure of risdiplam, either as the oral solution or tablet.³

Furthermore, 2-year data of risdiplam as an oral solution showed improvements in key motor milestones, with patients being able to swallow and feed orally and demonstrate cognitive skills without requiring permanent ventilation. All patients (with 3 or more SMN2 copies) achieved standing and walking milestones, sitting milestones (2 SMN2 copies), and 60% could stand and walk (2 SMN2 copies).⁴

Furthermore, there were no deaths or adverse events that led to withdrawal or treatment discontinuation. Common adverse events included teething, gastroenteritis, diarrhea, eczema, and pyrexia.³

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REFERENCES

1. FDA approves Roche’s Evrysdi tablet as first and only tablet for Spinal Muscular Atrophy (SMA). News release. Roche. February 12, 2025. Accessed February 12, 2025. https://www.globenewswire.com/news-release/2025/02/12/3025259/0/en/FDA-approves-Roche-s-Evrysdi-tablet-as-first-and-only-tablet-for-Spinal-Muscular-Atrophy-SMA.html

2. Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants. ClinicalTrials.gov identification: NCT04718181. Updated March 13, 2023. Accessed February 12, 2025. https://clinicaltrials.gov/study/NCT04718181?tab=results

3. Kletzl H, Heinig K, Jaber B, et al. Bioequivalence and food effect assessment for a new risdiplam tablet formulation in healthy volunteers. MDA Clinical and Scientific Conference. Accessed February 12, 2025. https://www.mdaconference.org/abstract-library/bioequivalence-and-food-effect-assessment-for-a-new-risdiplam-tablet-formulation-in-healthy-volunteers/

4. Majority of children with spinal muscular atrophy (SMA) treated with Roche’s Evrysdi are able to sit, stand and walk independently, two-year data demonstrate. News release. Roche. October 13, 2024. Accessed February 12, 2025. https://www.roche.com/media/releases/med-cor-2024-10-14