PTC Therapeutics Continue To Fight For Duchenne Muscular Dystrophy Community

Even after the third denial from the U.S. FDA, Food and Drug Administration, PTC Therapeutics Incorporation will not stop fighting for the drug Translarna that they believe will help patients suffering from Duchenne muscular dystrophy, originally reported by Bio Space. The drug is known to be a restorative therapy for proteins, which enables patients with genetic diseases to develop strong, functioning proteins. While the drug was successfully approved in Europe, PTC received their third rejection just this last October from the FDA.
Patients with Duchenne muscular dystrophy (DMD) face deterioration of muscle, which leads to limited movement and often paralysis. The disease is a severe degenerative neuromuscular disease that is caused from a mutated gene that affects the function of dystrophin (protein function). The condition is typically fatal and most patients die before they see their thirties. To read more about DMD, click here.

The FDA requested additional information on the drug from another trial, yet that seemed to only upset PTC. Stuart Peltz, the Chief Executive Officer of PTC is hoping to come back with more updates as they continue their process. Peltz shares the drug has been successful in Europe and is hopeful that the FDA is swayed in the near future. It has happened before, and three times recently, that the FDA wet back on their word and gave approval for drugs. Peltz hopes that the truth within the data does more fighting for them and eventually changes their thoughts.

While PTC wants to see more success with Translarna, they are continuing to make noise in the rare disease community. PTC took charge of Emflaza, and were able to get it into the hands of people who needed it. They are also in the process of developing a new treatment called RG7916 for SMA, spinal muscular atrophy. The new year is looking strong for PTC, as they’ve garnered nearly $174 million dollars this last year for Emflaza and Translarna alone. They anticipate to bring in even more, looking at a projected revenue of $170-$185 million this upcoming year.

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