NICE, the National Institute for Health and Care Excellence, has announced in its draft guidance it will not recommend funding on the NHS for breakthrough drug Translarna (ataluren) which could keep boys such as Harry Barnley from Weybourne School, who suffers from life-limiting condition, Duchenne muscular dystrophy, walking for longer.

Ahead of confirming its final position in the coming months, NICE has asked the company (PTC Therapeutics) to provide further justification for the cost of the drug and further clarification on the size of the benefit it provides in the treatment of Duchenne muscular dystrophy.

In August 2014, Translarna became the first drug addressing the cause of Duchenne muscular dystrophy to be approved in the EU and has been available in several EU countries since last year.

In the UK alone there are around 2,500 children and adults living with the condition, with the majority being male. It is caused by lack of dystrophin, a vital muscle protein, which leads to muscles weakening and wasting over time and increasingly severe disability.

The parents of Weybourne Infants School pupil Harry Barnley – who was diagnosed with the muscle-wasting condition in June 2013 – have been fighting endlessly to get funding for Translarna approved.

Without this drug, Harry could be left wheelchair-bound by the age of 10. To raise awareness and funds for Duchenne, parents Sue and Tom Barnley, set up the Help4Harry campaign which recently raised £12,000 through their fundraising night at Farnham Rugby Club.

Sue said: “To say we are devastated at this result is an understatement, this is a massive blow for families like ours. Harry needs this drug as soon as possible we don’t have days, weeks, months to wait, boys are losing the ability to walk while further delays and meetings happen. Living with Duchenne is hard enough but to be constantly battling for a drug that is known to slow the progression down is heartbreaking. We have to keep fighting.”

Commenting on the draft guidance, Professor Carole Longson, NICE Health Technology Evaluation Centre Director, said: “We are disappointed not to be able to recommend Translarna in this draft guidance. DMD is one of the most common and severe forms of muscular dystrophy. It reduces life expectancy and causes debilitating symptoms that severely affect the quality of life of people with the condition and their families and carers.”

After considering the evidence, and the opinions of the clinical and patient experts, the committee agreed that Translarna represents an important development in the treatment of DMD and could potentially prolong the time before children have to use a wheelchair, compared with best supportive care.

Professor Longson stated that the committee was not convinced that the proposed cost of Translarna was justified by the evidence presented on the additional health benefits associate­d with ataluren (Translarna) over standard therapy. Therefore, on the basis of the current evidence, the committee was minded not to recommend it for treating nonsense mutation DMD.

Robert Meadowcroft, chief executive of Muscular Dystrophy UK, said: “The news is extremely disappointing and a bitter blow for all of us awaiting the news. Having spoken to some of the families affected, it really is impossible to describe their heartbreak, particularly as just last week Lothian NHS Trust approved the drug for a family in Scotland.

“Many parents across the rest of the UK are watching their child weaken and lose mobility day by day, as this really is a race against time. If we are not careful, it will be too late for many of these boys to even be eligible to take Translarna.

“We must take every opportunity to protect children and their quality of life. The chance to be able to walk for longer can be immeasurably precious. We may not yet be able to halt the difficult physical and emotional challenges these children face, but with this drug we have the chance to delay them.”

NICE has not yet issued final guidance to the NHS, and the decision could change after further consultation.