The National Institute for Health and Care Excellence (NICE) has published the final guidelines for recommending Ataluren (Translarna) drug to treat Duchenne Muscular Dystrophy in Children below the age of five years.
Duchenne Muscular Dystrophy is a type of X-linked muscular dystrophy which causes degeneration of muscles and premature death. It usually affects boys and is mainly caused due to a dystrophin gene mutation in the X chromosome.
‘Ataluren, an innovative drug for the treatment of Duchenne Muscular Dystrophy in children will be made available on the NHS.’
The NICE followed by the announcements of National Health Services England and PTC therapeutics company has confirmed the 'managed access agreement' which provides the financial and clinical details of the drug Ataluren for Muscular Dystrophy.This agreement could benefit about 50- 60 children with muscular dystrophy.
The Chief Executive of NICE, Sir Andrew Dillon stated that the innovative drug Ataluren is the first of its kind which aims to treat the root cause of the disease and also benefits patients from the cruel disease that has very little treatment options. He also added that the five year managed access agreement between NHS England and PTC therapeutics will benefit children with muscular dystrophy as they will be able to access the drug as well as gather data on the drug efficacy before reviewing the guidelines and making decisions on whether to continue funding after five years.
Ataluren is a part of NICE's Highly Specialized Technologies Program which looks out for the treatment of rare diseases. The list price of the drug was found to be $ 290,000 per year.
The guidelines of NICE recommends Ataluren to be made available under the terms and conditions of Marketing Authorization Application and also the company should supply the drug as per the discount agreed in the patient access scheme.
This agreement is welcomed by muscular dystrophy charities in UK and the drug will be available in the NHS for children within few weeks.