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Evrysdi™ Approved in Japan for the Treatment of Spinal Muscular Atrophy
- 48th country that approved Evrysdi - - First commercial sale in Japan will trigger $10M milestone payment from Roche - SOUTH PLAINFIELD, N.J., June 23, 2021
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- 48th country that approved Evrysdi -\n - First commercial sale in Japan will trigger $10M milestone payment from Roche -\n\n\nSOUTH PLAINFIELD, N.J., June 23, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced 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 $10M milestone payment to PTC from Roche under its License and Collaboration agreement. \n\"We are thrilled to see the Evrysdi approval in Japan and are excited that SMA patients have an effective and convenient at home, oral treatment option that avoids the need for in-hospital administration,\" said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. \"The rapid adoption of Evrysdi globally underscores the importance of this drug and the profound benefit this therapy provides to SMA patients.\" \nEvrysdi is 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 United States by Genentech, a member of the Roche Group.\nAbout Spinal Muscular Atrophy (SMA)Spinal muscular atrophy (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. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to progressive muscle weakness over time. Depending on the type of SMA, an individual's physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.\nAbout Evrysdi™ (risdiplam)Evrysdi...