PPMD is pleased to learn that the FDA has acknowledged the filing over protest of PTC's New Drug Application (NDA) for Translarna™ (ataluren). PTC is seeking approval to market the drug for the treatment of nonsense mutation Duchenne muscular dystrophy patients in the United States. Translarna received marketing authorization for patients with nonsense mutation Duchenne muscular dystrophy in the European Union in August 2014 and is now available in over 25 countries. The FDA has granted standard review and assigned a Prescription Drug User Fee Act (PDUFA) date of October 24, 2017.
Read the Announcement from PTC Therapeutics:
PTC Therapeutics Announces FDA Acknowledgment of New Drug Application Filing for Translarna™ for the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy
- FDA has assigned a PDUFA date of October 24, 2017 -
SOUTH PLAINFIELD, N.J., March 6, 2017 /PRNewswire/-- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has acknowledged the filing over protest of PTC's New Drug Application (NDA) for Translarna™ (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The Company is seeking approval to market the drug for the treatment of nmDMD patients in the United States. Translarna received marketing authorization for patients with nmDMD in the European Union in August 2014 and is now available in over 25 countries.
The FDA has granted standard review and assigned a Prescription Drug User Fee Act (PDUFA) date of October 24, 2017. The PDUFA date is the target date for the FDA to complete its review of the NDA.
"We look forward to working closely with the FDA and the DMD community to bring this much-needed therapy to patients," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "We believe that the totality of clinical data in our NDA, which includes the results of two of the largest placebo-controlled DMD clinical trials ever conducted, demonstrates Translarna's benefits to patients and merits a full and fair review by the FDA, including an advisory committee meeting."
Primarily affecting males, Duchenne muscular dystrophy is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with DMD lose the ability to walk in their early teens and experience life-threatening lung and heart complications in their late teens and twenties. It is estimated that nonsense mutations account for approximately 13% of DMD cases.
PTC used the FDA's file over protest regulations to file the NDA. These regulations allow a company to have its NDA filed and reviewed following receipt of a refuse to file determination.
About Translarna™ (ataluren)
Translarna, discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Translarna is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Translarna is an investigational new drug in the United States. The development of Translarna has been supported by grants from Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA's Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.
About PTC Therapeutics
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary small molecule drugs targeting an area of RNA biology we refer to as post-transcriptional control. Post-transcriptional control processes are the regulatory events that occur in cells during and after a messenger RNA, or mRNA, molecule is copied from DNA through the transcription process. PTC's internally discovered pipeline addresses multiple therapeutic areas, including rare disorders and oncology. PTC has discovered all of its compounds currently under development using its proprietary technologies. PTC plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. For more information on the company, please visit our website www.ptcbio.com.