The NHS has dealt a “harsh blow” to children with a severe muscle-wasting disease by turning down early funding for a drug that could keep them able to walk longer, a charity has said.
Families of young boys with Duchenne muscular dystrophy have been waiting since last summer to hear whether NHS England will fund Translarna.
But they learned today that it has deferred its decision to the National Institute for Health and Care Excellence (Nice), which will not report back until early next year.
Many of the youngsters who could benefit from it are unlikely to be able to walk by then.
Robert Meadowcroft, chief executive of Muscular Dystrophy UK, said: “This news is a harsh blow to each and every boy and young man living with Duchenne muscular dystrophy.
“Translarna is the first drug licensed and available in the EU to treat the underlying genetic cause for a proportion of boys with this devastating condition.
“Parents of young children have faced a gruelling nine-month wait for an answer on funding, only to hear ‘no’ from NHS England.
“They have endured delays, confusion and been completely and utterly let down by NHS England on what should have been an urgent and compassionate decision.
“While boys in many EU countries have access to this treatment, boys here are now denied access to the only available treatment after months of being kept in suspense.”
The drug, the first to treat the disease which causes severe disability and cuts life expectancy to around 30, is already available in European countries such as Germany, Spain, France and Italy.
A mother whose 10-year-old son hoped to be able to take it said the decision was “heartbreaking”.
Archie Hill, who along with a number of other young boys went to Downing Street last month to deliver a letter to UK Prime Minister David Cameron in an effort to secure his support, would have been eligible for Translarna.
Louisa Hill, from Chalfont St Peter, Buckinghamshire, said: “We have fought so hard for this drug, for Archie, and for all those other boys that it could help.
“Archie is only 10. Living with Duchenne muscular dystrophy already gives him so much to deal with and it's only going to get tougher.
“We want to give Archie as much time as possible to just get on with being a child.”
Last month, during Prime Minister’s Questions, Cameron said he recognised “how vital it is to give those affected and their families a decision as soon as possible” but refused to answer whether the drug would be granted.
The delay in the decision comes after the NHS held a consultation earlier this year about how to approve drugs for rare conditions, causing similar frustrating waits to approve other crucial drugs.
Translarna costs around the same as current drugs to treat cystic fibrosis, Muscular Dystrophy UK said.