PTC Therapeutics Says Evrysdi Approved In Japan To Treat Spinal Muscular Atrophy - Quick Facts

(RTTNews) - PTC Therapeutics, Inc. (PTCT) announced Wednesday that Evrysdi(risdiplam) was approved in Japan by the Japanese Ministry of Health, Labor and Welfare for the treatment of spinal muscular atrophy (SMA).

The approval has been granted based on data from two pivotal studies evaluating Evrysdi in infants and adults living with type 1, 2 or 3 SMA. The first commercial sale will trigger a $10 million milestone payment to PTC from Roche under its License and Collaboration agreement.

Evrysdi is a survival motor neuron 2 (SMN2)-directed RNA splicing modifier designed to treat SMA by increasing and sustaining the production of SMN protein, which is found throughout the body and is critical for maintaining healthy motor neurons and movement.

Evrysdi has been approved for the treatment of patients with SMA, aged 2 months and older by the FDA and the EMA. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. Evrysdi is marketed in the U.S. by Genentech, a member of the Roche Group.

SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately 1 in 10,000 babies and when untreated is the leading genetic cause of infant mortality.