PTC Therapeutics Announces Positive Data from its Translarna Phase II Clinical Trial

PTC Therapeutics, Inc. announced the presentation of data from the Translarna (ataluren) Phase II Study 030 demonstrating that the safety and pharmacokinetic profile of Translarna in children from two to five years with nonsense mutation Duchenne muscular dystrophy (nmDMD) was consistent with that for older children.  Importantly, the data also showed that treatment with Translarna resulted in improvements in timed function tests and the North Star Ambulatory Assessment from baseline at weeks 28 and 52, with mean changes showing as much as a 25 percent improvement after one year. The data at 28 weeks formed the basis of the recent positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to expand the current indication of Translarna to include nmDMD ambulatory children from two to five years of age.  The data was presented at the International Congress on Neuromuscular Diseases in Vienna.

Translarna is the only approved treatment to address the underlying cause of nmDMD, a rare, genetic, muscle-wasting disease, and is currently licensed in Europe for ambulatory patients aged five years and older. “We are excited to demonstrate that Translarna showed an improvement over one year of treatment in patients with nonsense mutation Duchenne as young as two years of age,” stated Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics, Inc. “Irreversible muscle damage starts before the age of five. Early intervention is critical to maintain muscle function and delay disease progression.” An interim analysis of Study 030 demonstrated that at week 28, the safety and pharmacokinetic profile for Translarna in children aged two to five years is consistent with that for older children. Clinical benefits were also observed at 28 weeks with Translarna, with decreases versus baseline in the time to run/walk 10 meters, climb four stairs, and stand from lying face up (supine). The most common adverse events included pyrexia, ear infection, and nasopharyngitis. – Medical Buyer Bureau