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NHS England Enables Access to Translarna™ (ataluren) For Patients with Nonsense Mutation Duchenne Muscular Dystrophy

July 7, 2016

Important decision allows reimbursed access to Translarna, the first approved therapy to treat the underlying cause of Duchenne muscular dystrophy

SOUTH PLAINFIELD, N.J., July 7, 2016 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company and NHS England have successfully negotiated a Managed Access Agreement (MAA) for Translarna (ataluren) for ambulatory patients aged five years and older with nonsense mutation Duchenne muscular dystrophy (nmDMD). This decision provides reimbursed patient access to Translarna in England via a five-year MAA. Translarna previously received a positive recommendation from the National Institute for Health and Care Excellence (NICE) in April of 2016, subject to PTC and NHS England finalizing the terms of the MAA. NICE is expected to issue final guidance later this month following execution of the MAA, with implementation soon after.

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.”

PTC and NHS England have now finalized the outstanding aspects of the MAA which include a confidential financial arrangement and the collection of further data on the efficacy of Translarna for the treatment of nmDMD over a five-year period with NICE guidance to be reviewed again at the end of that period, before future funding decisions are taken.

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’sOffice 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].