The UK National Institute for Health and Care Excellence (NICE) has issued final guidance recommending US firm PTC Therapeutics’ Translarna (ataluren) to treat ambulatory patients with nonsense-mutation Duchenne muscular dystrophy (nmDMD).
Translarna (ataluren), which can be used to treat nmDMD patients of five years of age and above, successfully meets the requirements of the Managed Access Agreement (MAA) with National Health Service (NHS) England.
Action Duchenne CEO Paul Lenihan said: “We are delighted that this positive recommendation from NICE has now been published.
“This is such 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.”
Duchenne muscular dystrophy (DMD) mainly affects males and is a progressive muscle disorder caused by the lack of functional dystrophin protein that is essential to maintain the structural stability of skeletal, diaphragm and heart muscles.
Patients suffering from DMD lose the ability to walk from as early as ten years of age and are affected with fatal lung and heart complications during their late teens and early twenties.
PTC Therapeutics UK and Ireland vice-president and general manager Janis Clayton said: “Translarna is the first and only licensed treatment to address the underlying cause of Duchenne muscular dystrophy.
“I am very pleased that the completion of this important review process undertaken by NICE and NHS England now ensures a much-needed therapy is available for the children and young adults with nonsense-mutation DMD that meets the requirements of the MAA.”
In August 2014, Translarna (ataluren) was granted marketing authorisation by the European Commission (EC) to treat patients with nmDMD.
The drug is currently under annual review by the European Medicines Agency (EMA) with an opinion on renewal expected by the middle of this year.