Chugai Wins Approval For New Evrysdi Treatment In Pre-Symptomatic SMA Patients

Chugai Pharmaceutical Co., Ltd. has announced that the Ministry of Health, Labour and Welfare (MHLW) has granted approval for "Evrysdi® Dry Syrup 60 mg" (risdiplam) to be used for pre-symptomatic spinal muscular atrophy (SMA) identified through genetic testing. Additionally, a new dosage has been approved for infants under two months old. Evrysdi, which was designated as an orphan drug for spinal muscular atrophy in March 2019, received this approval following a priority review process.

The approval is grounded in findings from the international Phase II RAINBOWFISH trial, which focused on infants with pre-symptomatic SMA (up to 6 weeks old at the time of their first dose) who were genetically diagnosed with the condition. The study involved infants with two or more copies of the SMN2 gene, where fewer copies typically indicate a more severe form of the disease.

Since motor neuron degeneration in SMA can start before visible symptoms appear, early intervention is crucial to improve outcomes. Newborn screening plays a vital role in achieving timely diagnoses. Evrysdi is expected to offer significant medical benefits by allowing treatment to begin immediately after diagnosis, even before symptoms emerge.

Chugai’s President and CEO, Dr. Osamu Okuda,stated, “We are very pleased that with this approval, we can now deliver Evrysdi to people with SMA of all ages after birth, regardless of symptom onset. As the only oral treatment for SMA, Evrysdi has been helping many people with SMA and their families. SMA treatment is expected to maximize its effect when intervention begins at an earlier stage. We are confident that this approval will allow Evrysdi to contribute even more to SMA treatment.”