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PTC Therapeutics Announces Commercial Milestone Payment Following the Recent European Approval of Evrysdi™
SOUTH PLAINFIELD, N.J., April 1, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that a $20 million milestone payment was triggered
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"SOUTH PLAINFIELD, N.J., April 1, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that a $20 million milestone payment was triggered by the first commercial sale of Evrysdi™ (risdiplam) in the European Union under its License and Collaboration Agreement with Roche. Approval for Evrysdi from the European Medicines Agency was received on March 30 for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older. \n\"We are happy to see the rapid adoption of Evrysdi in the EU which speaks to the need for new treatments for SMA patients,\" said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. \"We are delighted that an effective at-home therapy will be available to SMA patients. We recognize that a large proportion of SMA patients in the EU are currently not receiving an approved therapy.\"\nRoche is working closely with reimbursement and assessment bodies in European countries to enable broad and rapid access to SMA patients. Evrysdi is immediately accessible to patients in Germany and will be accessible from early April to patients in France through the cohort Temporary Authorization for Use. Evrysdi has currently been approved in 38 countries and submitted for Health Authority review in a further 33 countries.\nEvrysdi is based on PTC science and is commercialized in the United States by Genentech, a member of the Roche Group. Roche led the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.\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™ (risdiplam) is a survival motor neuron 2 (SMN2)-directed RNA spl...