PTC Therapeutics, Inc. (PTCT - Free Report) announced that it has initiated the process of a rolling submission of a new drug application (NDA) to the FDA for its Duchenne muscular dystrophy (DMD) drug, Translarna. The company is evaluating Translarna for the treatment of nonsense mutation DMD (nmDMD).
We note that a rolling submission will allow the company to complete portions of the application which needs to be submitted and reviewed by the FDA on an ongoing basis. PTC Therapeutics expects to complete the application in the fourth quarter of 2015 after the completion of the confirmatory phase III study on Translarna in nmDMD patients.
We remind investors the company had received conditional marketing approval for Translarna in the EU for the treatment of nmDMD ambulatory patients (five years of age and older) in August this year. A conditional approval makes it necessary for the company to complete phase III study in order to gain full approval. Earlier this month, the company announced that it has launched Translarna in Germany.
We are encouraged by the initiation of the rolling NDA submission of Translarna by the company. According to the press release issued by the company, nonsense mutation is estimated to be the cause of DMD in approximately 13% of patients or approximately 2,000 patients in the U.S. and 2,500 patients in the EU.
Currently, companies like Prosensa and Sarepta Therapeutics (SRPT - Free Report) are developing therapies for the treatment of DMD.
Meanwhile, PTC Therapeutics is also evaluating Translarna in several additional indications including nonsense mutation cystic fibrosis and mucopolysaccharidosis type I.
We expect investor focus to remain on updates pertaining to Translarna.
PTC Therapeutics currently carries a Zacks Rank #3 (Hold). A better-ranked stock in the health care sector is Biodel Inc. carrying a Zacks Rank #1 (Strong Buy).