After months of delays, and despite a prominent campaign, NICE has announced it will not recommend funding on the NHS for breakthrough drug Translarna which could keep boys with life-limiting Duchenne muscular dystrophy walking for longer.
Ahead of confirming its final position in the coming months, NICE have asked the company to provide further justification for the cost of the drug.
In August 2014, Translarna became the first drug addressing the cause of Duchenne muscular dystrophy to be approved in the EU. It has been available in several EU countries since last year.
Families of the boys affected have spoken of their anger at the news.
“We are absolutely devastated. How do we tell Archie he is not allowed a drug that will keep him walking and living for longer because NHS England and drug companies cannot agree on a price.
“Today's 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."