Roche's Risdiplam Tablets Approved in China for Spinal Muscular Atrophy Treatment

On August 27, Roche announced that China's NMPA has officially approvedEvrysdi(General Name: Risdiplam)TabletLaunched for the treatment ofSpinal Muscular Atrophy in pediatric and adult patients aged 2 years and above and weighing ≥20 kg(hereinafter referred to as SMA)。

It is reported that the new 5 mg tablet does not require refrigeration and can be stored at room temperature. Patients can swallow the tablet whole or disperse it in water, with or without food.

Screenshot source: Roche Official WeChat

SMA is a systemic, multi-system progressive neuromuscular genetic disease. Due to the deletion or mutation of the SMN1 gene, SMA patients cannot produce enough functional SMN protein, affecting important functions such as movement, breathing, and swallowing. As the disease progresses, SMA patients lose their mobility, experience respiratory and swallowing difficulties, and may face life-threatening conditions.

Risdiplam is a SMN2 gene double-site splicing modifier that targets the direct cause of SMA, providing a lasting and stable increase in SMN protein levels. Risdiplam can penetrate the blood-brain barrier, simultaneously enhancing and stabilizing SMN protein levels in both the central and peripheral systems, leading to continuous improvement in patients' motor function and increased survival rates.

Previously,Risdiplam Oral Solution PowderApproved in China in 2021 for the treatment of SMA patients aged 2 months and above,Is the first approved oral treatment drug for SMA in ChinaIn 2023, the drug was included in the national medical insurance catalog for the first time through medical insurance negotiations, and was approved in the same year to expand its applicable population to SMA patients aged 16 days and above.

The recent approval of Risdiplam tablets in China is expected to further enhance patients' independence and adherence to medication, improving the long-term management of SMA.

Outside of China, risdiplam tablets have been successively approved in the United States in February 2025 and in the European Union in June 2025, primarily based on the results of the bioequivalence study NCT04718181. The research data shows,Whether swallowed whole or dispersed in water, its exposure level in the body is equivalent to that of the oral solution formulation.This indicates that the efficacy and safety available to patients taking the tablet form are comparable to the current standards of the oral solution formulation.