Drug to treat type of muscular dystrophy in children expected to get green light


A drug to treat a type of muscular dystrophy in children - which captured the attention of Prime Minister David Cameron -  is expected to be given the green light for use on the NHS.

The National Institute for Health and Care Excellence (Nice) will publish guidance on Monday relating to Translarna (also called ataluren).

In February, health minister George Freeman confirmed NHS England was in discussions with pharmaceutical company, PTC Therapeutics, on a "managed access agreement" for the NHS in England to fund Translarna.

Muscular Dystrophy UK is also hopeful such an agreement can be reached and has said the drug could transform young children's lives.

Translarna is for patients with a particular type of Duchenne muscular dystrophy, which is one of the most common and severe forms of the disease.

Duchenne usually affects boys in early childhood. They generally only live into their 20s or 30s.

Translarna could help around 10 to 15% of those affected by Duchenne in childhood and has been shown to slow down progression of the disease.

Last year, Archie Hill, of Gerrards Cross, Buckinghamshire, had his wish come true when he was able to hand a petition in person to Mr Cameron.

The Prime Minister surprised Archie and his family outside 10 Downing Street as they handed in a 22,785 signature petition calling on the NHS to end delays to offering Translarna.

Mr Cameron was also asked in Parliament about the drug by Archie's local MP, Cheryl Gillan.

He said the decision should be left up to "clinicians rather than politicians" but called on industry to find a way to lower the cost of some drugs.

Archie, who was diagnosed with the severe and progressive Duchenne at the age of three, has also had his plight backed by England and Arsenal footballer Jack Wilshere.

Wilshere, who is an ambassador for Muscular Dystrophy UK, has called on the NHS to fund the drug, which can delay the need for children to use a wheelchair.

Most youngsters with the condition become reliant on a wheelchair before they reach the age of 12.

In October, Nice said it was minded not to approve Translarna but asked PTC Therapeutics to provide further information on cost-effectiveness and the results of a clinical trial of the drug.

It has been considering the evidence since then and will report its view on Monday.

In 2014, Translarna became the first genetic therapy for Duchenne muscular dystrophy to be approved within the EU. It is available in several other European countries.

Earlier this week, the Scottish Medicines Consortium (SMC) said it would not approve the drug for use in Scotland.