Health Canada Initiates Review of PTC Therapeutics' New Drug Submission For Translarna™ for the Treatment of Duchenne Muscular Dystrophy

If approved, Translarna would be the first treatment available to patients in Canada that addresses an underlying cause of Duchenne muscular dystrophy

BURLINGTON, ON, Sept. 18, 2015 /CNW/ - PTC Therapeutics International, Ltd., a subsidiary of PTC Therapeutics, Inc. (NASDAQ: PTCT), today announced that Health Canada has initiated their review of the company's New Drug Submission (NDS) for Translarna™ (ataluren) to treat nonsense mutation dystrophinopathy.  The NDS will be reviewed with advance consideration under the Health Canada policy of Notice of Compliance with Conditions (NOC/c). 

If approved, Translarna would be the first treatment available to patients in Canada that addresses an underlying cause of dystrophinopathy, including nonsense mutation Duchenne muscular dystrophy (nmDMD). 

"We now have a better understanding about the different kinds of genetic mutations that can cause Duchenne muscular dystrophy and are making great progress in developing potential treatments that address the underlying genetic causes of the disease," said Dr. Craig Campbell, Head of Pediatric Neurology at London Health Sciences Centre. "Every day matters for patients with nonsense mutation Duchenne muscular dystrophy. We look forward to the potential for a new treatment option that addresses an underlying cause of this relentlessly progressive disease which leads to the loss of walking ability in childhood, and premature death."

Health Canada's NOC/c policy is available for promising new drug therapies intended for the treatment, prevention or diagnosis of serious, life-threatening or severely debilitating diseases, or conditions for which no drug is presently marketed in Canada, or that represents a significant improvement in the benefit/risk profile over existing therapies.  An NDS reviewed under the advanced consideration NOC/c policy is granted an expedited review in 200 days compared to standard NDS review of 300 days.

"There is a clear and urgent need for a treatment for people living with nonsense mutation Duchenne muscular dystrophy," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "We are grateful that Health Canada has recognized this need by granting Translarna an expedited review."

"This filing is a very important milestone for us and we look forward to working with Health Canada to bring Translarna to patients in Canada as soon as possible," said Michael Cloutier, General Manager of PTC Therapeutics in Canada.  "We are very pleased to have a new Canadian presence with our office in Burlington, Ontario, and are now fully operational in Canada."

The NDS for Translarna is based on the safety and efficacy results from a randomized double-blind multicenter study in 174 nmDMD patients for 48 weeks, and an additional retrospective analyses of study data. These data formed the basis for PTC's marketing authorization application in Europe in 2014, which resulted in Translarna being licensed in the European Economic Area for the treatment of nmDMD in ambulatory patients aged five years and older.  PTC Therapeutics is conducting a confirmatory Phase 3 randomized double-blind multicenter study in patients with nmDMD and the top line results from this study will be available in the fourth quarter of 2015.

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. The safety and efficacy of Translarna is still under investigation in Canada, and Canadian market authorization has not yet been obtained. 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 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, the more severe form of the disorder, 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 a nonsense mutation is the cause of DMD in approximately 13 per cent of patients.

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

Forward Looking Statements:
This release contains forward-looking statements within the meaning of The U.S. Private Securities Litigation Reform Act of 1995. All statements, other than those of historical fact, contained in this release, including statements regarding the future expectations, plans and prospects for PTC are forward-looking statements. Other forward-looking statements may be identified by the words "plan," "guidance," "anticipate," "believe," "estimate," "expect," "intend," "may," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including the factors discussed in the "Risk Factors" section of PTC's most recent periodic report (Form 10-K or Form 10-Q) filed with the Securities and Exchange Commission as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this release except as required by law.

SOURCE PTC Therapeutics

For further information: Investors: Emily Hill, +1 (908) 912-9327,; Media: Jane Baj, +1 (908) 912-9167,

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