Business
PTC Therapeutics Announces FDA Approval of Evrysdi™ (risdiplam) for the Treatment of Spinal Muscular Atrophy in Adults and Children 2 months and older
- Evrysdi represents the first at home, oral treatment approved for infants, children and adults with all SMA types- - Marketing Authorization Application
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- Evrysdi represents the first at home, oral treatment approved for infants, children and adults with all SMA types-\n - Marketing Authorization Application submission to European Medicines Agency is imminent -\n - Risdiplam is the first approved therapy discovered and developed from PTC Therapeutics' proprietary splicing platform -\n\n\nSOUTH PLAINFIELD, N.J., Aug. 7, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has approved Evrysdi™ (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older. Evrysdi showed clinically meaningful improvements in motor function and obtainment of developmental milestones across two trials in patients age two months and above and across all levels of disease severity, including types 1, 2, and 3 SMA. Infants achieved key motor milestones not normally seen in the natural course of the disease, such as the ability to sit without support. Evrysdi also preserved vital functions and improved survival at 12 months. In particular, Evrysdi improved survival without permanent ventilation compared to natural history when measured at 12 and 23 months. \n\"Today marks an incredibly important moment for the broader SMA patient community that had been in dire need of safe and effective treatment options,\" said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. \"We are appreciative of the patients, physicians and others involved with its development, and our partners at the SMA Foundation and Roche. We are proud that the first oral treatment for spinal muscular atrophy patients was discovered through our splicing platform. I believe that this is one of the most innovative products in the pharmaceutical industry and we are gratified to have played a key role in its discovery and development.\"\nEvrysdi has been studied in a robust clinical trial program in SMA with more than 450 patients and subjects. The program includes infants aged 2 months to adults aged 60 with a range of symptoms and motor function including patients with scoliosis or joint contractures. Patients previously treated for SMA with other medications were also included. The approval is based on results from two clinical studies designed to represent a broad spectrum of pati...