The National Institute for Health and Care Excellence (NICE) has recommended PTC Therapeutics, Inc.’s Translarna™ (ataluren) for ambulatory patients aged five years and older with nonsense mutation Duchenne muscular dystrophy (nmDMD) in connection with a Managed Access Agreement (MAA) with NHS England. The provision of patient access is subject to the finalization of the NICE draft guidance, which the agency expects in May of 2016.
“This is an important day for the Duchenne community, which has been working hard to make this ground-breaking drug available to boys with nonsense mutation Duchenne muscular dystrophy,” said Paul Lenihan, Chief Executive of Action Duchenne. “We are delighted by this positive recommendation and NICE’s recognition that Translarna is an important new medicine for patients. This decision is a hugely encouraging sign that both NICE and NHS England have listened to the patient community, bringing hope to each and every parent and patient fighting 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 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.
“We are extremely pleased by the NICE recommendation, which recognizes Translarna as an innovative medicine with the potential to change the course of this devastating disease,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “This is a crucial step for the boys and young men in England with nonsense mutation DMD. We are grateful to the patients, families, advocacy groups and physicians who have supported PTC Therapeutics through this important access process and look forward to working with NHS England to conclude the managed access agreement.”
PTC and NHS England are in the process of finalizing an MAA outlining financial and clinical details surrounding the use of Translarna, including a confidential financial arrangement. The MAA is expected to allow PTC to collect 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.
Translarna was approved by the European Commission in August 2014 to treat nmDMD and is currently available to patients in 23 countries through either expanded access programs or commercial sales.