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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 from as early as 10 years of age and experience life-threatening lung and heart complications in their late teens and early twenties.
"This is an important day in England for children and young adults suffering from DMD," said Stuart W. Peltz , Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "We are extremely pleased to have reached a successful outcome with NHS England, which will provide long-awaited access to Translarna for patients with nonsense mutation DMD. We are grateful to the patients, families, advocacy groups and physicians for their tremendous effort in supporting PTC Therapeutics throughout this important and rigorous access process."
Translarna received marketing authorization from the European Commission to treat nmDMD in August 2014 , which is currently under annual review by the European Medicines Agency with an opinion on renewal expected mid-2016. Translarna is currently available to patients in more than 20 countries through either expanded access programs or commercial sales.
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.
Further information about Translarna, including the European Public Assessment Report, Summary of Product Characteristics and Patient Information Leaflet, is available on the European Medicines Association website .
This medicinal product is subject to additional monitoring. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system or to PTC at [email protected] .
About PTC Therapeutics, Inc. 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 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 .
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Forward Looking Statements: All statements, other than those of historical fact, contained in this press release, are forward-looking statements, including statements regarding the: future expectations, plans and prospects for PTC; timing and outcome of PTC's regulatory strategy and process, including the execution of the MAA, issuance of final guidance from NICE with respect to Translarna, the implementation of such guidance in England , and the European Medicines Agency's (EMA) opinion with respect to the potential renewal or approval of the marketing authorization for Translarna in nmDMD in the European Economic Area (EEA) and any restrictions or conditions that may be placed on any such renewal or approval; PTC's ability to maintain its current marketing authorizations, including its ability to satisfy any obligations or conditions that may be placed on its marketing authorization in the EEA, or obtain and maintain additional marketing authorizations; the clinical utility and potential advantages of Translarna; PTC's ability to continue to supply Translarna to patients across Europe and in other territories; PTC's strategy, future operations, future financial position, future revenues or projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "predict," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.
As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that Translarna will receive full regulatory approval in any territory or maintain its current marketing authorization in the EEA, or prove to be commercially successful in general, or specifically with respect to the treatment of nmDMD.
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 press release except as required by law.
SOURCE PTC Therapeutics, Inc.