Business
PTC Therapeutics Announces CHMP Recommendation of Translarna™ (ataluren) Label Update for Non-Ambulatory Patients with Duchenne Muscular Dystrophy
- EU label update supports Translarna use in patients who became non-ambulatory while on therapy - SOUTH PLAINFIELD, N.J., June 29, 2020 /PRNewswire/ -- PTC
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- EU label update supports Translarna use in patients who became non-ambulatory while on therapy -\n\n\nSOUTH PLAINFIELD, N.J., June 29, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended by a majority of votes to remove the statement \"efficacy has not been demonstrated in non-ambulatory patients\" from the SmPC for Translarna™ (ataluren). This label change enables healthcare professionals to use their clinical judgement to make treatment decisions for their patients on Translarna who have lost ambulation.1 The change also should support reimbursement agencies granting continued access to Translarna for patients who become non-ambulatory during the course of their treatment. The CHMP's positive opinion is subject to final approval by the European Commission, which is normally granted in a two-month time frame.\n\"We are excited to see that the CHMP adopted the positive opinion for this label modification allowing patients who become non-ambulatory to continue to use Translarna,\" said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. \"All nonsense mutation Duchenne patients should be able to benefit from continued Translarna use, ensuring they have the best chance of preserving muscle function for as long as possible.\"\nTranslarna is the only treatment for the underlying cause of Duchenne caused by a nonsense mutation and works by restoring dystrophin production.1,2 It is approved by the EMA for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged two years and older. Real world evidence and clinical trials have demonstrated the potential of Translarna to slow disease progression and improve outcomes before and beyond loss of ambulation: \nIn a long-term, open-label extension study (Study 019), Translarna preserved lung function in non-ambulatory patients for an additional four years compared with patients treated with standard of care from a long-term natural history study (the Cooperative International Neuromuscular Research Group (CINRG) natural history database of Duchenne patients).3 Data from the STRIDE Registry, the first international drug registry for Duchenne patients receiving Translarna, demonstrated that boys ...