Date: Jan 08, 2016 Source: PR Newswire (
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SOUTH PLAINFIELD, N.J., Jan. 8, 2016 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the completion of its rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for Translarna™ (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). PTC has also fulfilled the principal requirement of the European Medicines Agency (EMA) in connection with its approval of Translarna in August 2014 by submitting the results of its Phase 3 ACT DMD clinical trial to the agency.
"The completion of these regulatory submissions for Translarna is a significant milestone for PTC," said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. "Following Translarna's approval within Europe and other countries as the first-ever treatment for an underlying cause of Duchenne muscular dystrophy, we are planning to bring Translarna to patients in the United States. We believe the results across multiple clinical studies demonstrate Translarna's ability to modify disease progression in nonsense mutation DMD and confirm its strong safety profile. We appreciate the dedication and support of the clinical investigators, patients and their families, and we look forward to working with regulators to continue to bring this much needed treatment to patients."
About Duchenne Muscular Dystrophy
Primarily affecting males, Duchenne muscular dystrophy (DMD) 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 as early as age 10 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, or approximately 2,000 patients in the United States.
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.
Translarna has the potential to benefit patients with genetic disorders caused by a nonsense mutation. On average, 11% of every monogenic disorder is caused by a nonsense mutation. PTC's strategy is to expand the clinical development of Translarna across multiple genetic disorders to deliver on the company's commitment to address rare and neglected disorders.
The FDA and the EMA have granted Translarna Orphan Drug status for the following indications: DMD, cystic fibrosis (CF), Mucopolysaccharidosis I (MPS I), and aniridia.
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, oncology and infectious diseases. 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
