PTC Therapeutics, Inc. Announces STRIDE Data Show Translarna Delays Loss of Ambulation by More Than Five Years in Boys with Nonsense Mutation Duchenne Muscular Dystrophy
September 20, 2021 at 08:30 am EDT
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PTC Therapeutics, Inc. presented real-world results from the STRIDE patient registry demonstrating that treatment with Translarna™ (ataluren) delays loss of ambulation by more than five years in boys with nonsense mutation Duchenne muscular dystrophy (nmDMD) compared to standard of care (SoC) alone. Pulmonary function decline was also delayed by 1.8 years in those treated with Translarna and SoC. A time-to-event analysis of five years of registry data, presented at the World Muscle Society (WMS) 2021 Virtual Congress, shows that boys treated with Translarna plus SoC had a median age of loss of ambulation of 17.9 years old compared with 12.5 years old for those on SoC alone. At 12 years old, 80% of boys receiving Translarna plus SoC are still walking, compared to 52% of the boys receiving SoC alone. The median age that boys treated with Translarna reached a predicted forced vital capacity (FVC) lower than 60% was 17.6 years old, compared with 15.8 years old for those who did not receive Translarna. This delay in loss of lung function is critical, as the sub-60% threshold is considered the milestone at which patients usually start to require respiratory physical therapy. The data was compared with natural history from a propensity-score matched cohort in a long-term natural history study (the Cooperative International Neuromuscular Research Group (CINRG) natural history database of Duchenne patients). Translarna continues to be well tolerated in nmDMD patients, and the safety results, representing 1059 patient-years of exposure, remain consistent with the therapy's known safety profile.
PTC Therapeutics, Inc. is a global biopharmaceutical company. The Company is focused on the discovery, development, and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. The Company has a diversified therapeutic portfolio that includes several commercial products and product candidates in various stages of development, including discovery, research and clinical stages, focused on the development of new treatments for multiple therapeutic areas for rare diseases relating to neurology and metabolism. It has two products, Translarna (ataluren) and Emflaza (deflazacort), for the treatment of Duchenne muscular dystrophy (DMD) a rare, life-threatening disorder. Its Upstaza, a gene therapy for the treatment of Aromatic L-Amino Decarboxylase (AADC) deficiency, a rare central nervous system (CNS) disorder. Its Tegsedi and Waylivra are for the treatment of rare diseases. Its Evrysdi, a treatment for spinal muscular atrophy (SMA).
PTC Therapeutics, Inc. Announces STRIDE Data Show Translarna Delays Loss of Ambulation by More Than Five Years in Boys with Nonsense Mutation Duchenne Muscular Dystrophy