PTC Therapeutics
Jul 9, 2014
PTC Therapeutics Announces Expanded Access Program For Translarna™ (ataluren)
PTC Therapeutics Announces Expanded Access Program For Translarna™ (ataluren) SOUTH PLAINFIELD, NJ – July 9, 2014 – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the initiation of a reimbursed expanded access program (EAP). PTC's EAP program is intended to make Translarna&#8482 (ataluren) available to patients before commercial availability in certain countries. Where mechanisms exist and in accordance with local regulations, PTC will make Translarna available to nonsense mutation Duchenne muscular dystrophy (nmDMD) patients through funded EAP programs.

Funded Named Patient Programs have already been authorized in Turkey and Spain. Today, the French National Agency for Medicines and Health Products Safety (ANSM), has granted a Temporary Authorization for Use (Autorisation Temporaire d'Utilisation de cohorte). Government allocations to hospitals will allow payment for Translarna for patients included in the ATU cohort program.

"For children and young men living with DMD, a rapidly progressing muscle wasting disorder, every day counts. We are committed to working with regulators, payors and the DMD community to enable Translarna to reach patients as soon as possible wherever reimbursed EAP mechanisms exist," stated Mark Rothera, Chief Commercial Officer of PTC Therapeutics, Inc.

ABOUT TRANSLARNATM (ATALUREN) Translarna, discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of 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. 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 (DMD) 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% of patients, or approximately 2,000 patients in the United States and 2,500 patients in the European Union. More information about DMD is available through the Muscular Dystrophy Association (, Parent Project Muscular Dystrophy (, Action Duchenne (, United Parent Projects Muscular Dystrophy (, Muscular Dystrophy Campaign ( and AFM (l'Association française contre les myopathies), (

ABOUT PTC THERAPEUTICS, INC. PTC is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are essential to proper cellular function. PTC's internally discovered pipeline addresses multiple therapeutic areas, including rare disorders, oncology and infectious diseases. PTC has developed proprietary technologies that it applies in its drug discovery activities and in collaborations with leading biopharmaceutical companies. For more information on the company, please visit our website



FORWARD LOOKING STATEMENTS: This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements, other than those of historical fact, contained in this release, are forward-looking statements, including statements concerning the future expectations, plans and prospects for PTC, the timing of regulatory approvals, including any determination (whether positive or negative) by the European Commission with respect to conditional marketing authorization for Translarna in nmDMD, the development of and potential market for Translarna, including our estimates regarding the size of the nmDMD patient population, our Phase 3 clinical trial for Translarna in nmDMD, including the timing of enrollment for such trial, our ability to satisfy the obligations necessary to qualify or continue to qualify for EAP programs or to obtain full approval for Translarna in nmDMD, and the objectives of management. Other forward-looking statements may be identified by the words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan" "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.

Our actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements we make as a result of a variety of risks and uncertainties, including among others, those related to our expectations for regulatory approvals, including the European Commission's determination with respect to conditional marketing authorization for Translarna in nmDMD, the initiation and conduct of clinical trials, availability of data from clinical trials, our scientific approach and general development progress, the availability or commercial potential of our product candidates, market acceptance by physicians, patients, third-party payors and others in the medical community necessary for commercial success, and the other factors discussed in the "Risk Factors" section of our most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission. You are urged to carefully consider all such factors. The forward-looking statements included in this press release represent our views only as of the date of this release, and we do 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 applicable law.