PTC Therapeutics Expands Global Presence in Support of Translarna™ Launch in Duchenne Muscular Dystrophy

PTC Therapeutics Expands Global Presence in Support of Translarna™ Launch in Duchenne Muscular Dystrophy

SOUTH PLAINFIELD, N.J., Sept. 17, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the expansion of its global presence with the addition of key executives to leadership positions in PTC's global commercial team and the establishment of its international headquarters in Dublin, Ireland. Significant pre-launch activities are well underway as PTC prepares for the commercial launch of Translarna™ (ataluren) in the European Union. In August of this year, Translarna received approval from the European Medicines Agency for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).

PTC has recently expanded its leadership team within the commercial organization with the appointment of Adrian Haigh as Senior Vice President & General Manager of the EMEA Region. Adrian joins PTC from Gentium SpA, where he held the position of Senior Vice President, Commercial Operations. While at Gentium, Adrian was responsible for building and managing the company's commercial and medical affairs organization. Prior to joining Gentium, Adrian served as Regional Vice President, Commercial Operations at Biogen Idec, where he managed several affiliates and also the global distribution business.

"We are delighted to be able to attract top talent such as Adrian to PTC's commercial organization," stated Mark Rothera, Chief Commercial Officer, PTC Therapeutics, Inc. "Adrian brings leading experience both in establishing European commercial organizations and in launching therapies for rare disorders. We have also appointed senior executives with excellent rare disease and commercial launch experience for key countries including Germany, the UK, France, Italy and the Nordic Region. We are thrilled to welcome this talented group to the PTC team. In addition, we have also established distribution and marketing partnerships with experienced companies in the EMEA region including Spain, Turkey and Israel. These appointments and the opening of our international headquarters in Dublin are clear and important steps forward in PTC's geographic expansion and its commitment to bring Translarna to all patients who may benefit globally."

"I am delighted to join PTC at this critical point in the evolution of the company," stated Adrian Haigh. "DMD is a devastating, life-threatening condition and my priority is to ensure that all patients who qualify for treatment with Translarna gain rapid access to this new therapy. PTC's unrelenting commitment to bring disease modifying therapies to patients suffering from rare disorders is impressive and I look forward to being part of that effort."

PTC's international headquarters in Dublin will serve as the central hub for the commercial launch of Translarna to take advantage of its location with respect to PTC's third party manufacturing and supply chain. In addition, PTC has begun establishing subsidiaries in each of the key European countries where Translarna is expected to initially become commercially available.

About Translarna™ (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. PTC has received conditional marketing authorization in the European Economic Area for Translarna 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 US. 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, 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 www.ptcbio.com.

PTC Therapeutics, Inc. 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 press release, including statements regarding the future expectations, plans and prospects for PTC; the timing of regulatory approvals; the development of and potential market for Translarna, including our plans for commercialization in various countries, the creation of subsidiaries, the establishment of a distribution network, and the entry in to relationships with third parties in certain countries; our ability to satisfy the obligations necessary to obtain full approval for Translarna in nmDMD either in the EU or elsewhere; and the objectives of management, are forward-looking statements. Other forward-looking statements may be identified by the words "will," "anticipate," "believe," "estimate," "expect," "look forward to," "intend," "may," "plan" "predict," "project," "target," "potential," "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; the initiation, conduct and results of clinical trials; the 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; our sales, marketing and distribution capabilities and strategy; 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 United States Securities and Exchange Commission. You are urged to carefully consider all such factors. In addition, 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.

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