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PTC Therapeutics Announces the European Approval of Evrysdi™ for the Treatment of Spinal Muscular Atrophy
- First and only at home treatment approved for SMA - - Proven efficacy in adults and children with SMA 2 months and older - SOUTH PLAINFIELD, N.J., March 30,
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- First and only at home treatment approved for SMA -\n - Proven efficacy in adults and children with SMA 2 months and older -\n\n\nSOUTH PLAINFIELD, N.J., March 30, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the European Commission (EC) has granted marketing authorization to Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy (SMA) in patients 2 months and older. Evrysdi will be for SMA Type 1, Type 2 or Type 3 patients with one to four SMN2 copies. The EC approval is based on two studies; 1) the efficacy results from the FIREFISH study in infants aged 2 to 7 months with symptomatic Type 1 SMA, and 2) the SUNFISH study in children and young adults with Type 2 or 3 SMA. The review was completed under the accelerated assessment pathway for medicines, which is offered to medicines deemed to be of major interest for public health and therapeutic innovation.\n\"We're thrilled that Evrysdi, with proven efficacy and safety, has been rapidly approved in Europe so that the SMA community will now have access to a much needed convenient at-home treatment,\" said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. \"Evrysdi was discovered using our novel splicing technology and is an incredibly innovative drug that was the first in demonstrating that a small molecule can selectively and specifically modulate splicing to treat the underlying cause of this devastating disease. We're excited to see this therapy become more available to patients around the world.\"\nMore than 3,000 patients have now been treated with Evrysdi in clinical trials, compassionate use programs and real-world settings, with patients ranging from birth to over 70 years old including those previously treated with other SMA therapies. The EU approval is applicable to all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein. Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation in 2019. Maintenance of Orphan Drug Designation was recently confirmed by the Committee for Orphan Medicinal Products based on the assumption of Evrysdi's significant benefit over existing treatments. Evrysdi has currently been approved in 38 countries and submitted in a further 33 countries.\nEvrysdi is based on PTC science and is commercialized in...