PTC Therapeutics, Inc. has just announced its novel protein restoration therapy Translarna™ (ataluren) is now available to patients with Duchenne Muscular Dystrophy (DMD) in Germany, which is the first European Union country to offer the drug. The first batch of shipments are expected to roll out this week. PTC Therapeutics is focused on discovering and developing breakthrough orally-stable, small molecule drugs that specifically act on post-transcriptional control processes.
Duchenne Muscular Dystrophy is a progressive muscle disorder wherein the body lacks functional dystrophin protein, which is essential to the structural stability of the skeletal, diaphragm, and heart muscles. DMD is commonly diagnosed in males, some losing the ability to walk as young as 10 years old, and some teenagers already experiencing potentially fatal respiratory and cardiac complications. Experts believe the lack of dystrophin in this condition is caused by a “nonsense mutation,” which Translarna has been observed to effectively address.
“The launch of Translarna in Germany marks another exciting milestone for the DMD community,” said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. “Through our reimbursed early access programs and commercial efforts, we are working hard to bring Translarna to those who may benefit from this drug. We remain committed to bringing Translarna to patients in Europe, and around the world, as quickly as possible. We know every day counts for the patients and their families.”
Germany is the first country to make the drug commercially available, soon after its Marketing Authorization approval from the European Commission earlier on August 2014 for ambulatory patients with nonsense mutation DMD, at least 5 years of age. Translarna has yet to submit an application to the US Food and Drug Administration, but PTC Therapeutics is already planning to seek approval in the US after a confirmatory Phase III trial is completed, which was announced fully enrolled on September 2014. Data from this study is expected in Q4 2015.
Last month, November 2014, PTC Therapeutics was selected to receive the Outstanding Innovation Award at The 2014 Rare & Orphan Advocacy & Research Awards for Translarna. The company received the recognition during the 5th Annual World Orphan Drug Congress Europe, which took place between November 12 and 14, in Brussels, Belgium.