Duchenne drug to be made available on NHS

The boys took their case to Downing Street Credit: ITV

For the first time ever, children will be able to access a drug tackling an underlying cause of life-limiting, Duchenne muscular dystrophy through NHS England. Translarna could keep children with the severe muscle-wasting condition walking for longer.

NICE has announced that it has secured a Managed Access Agreement on the drug.

NICE’s move comes just days after the Scottish Medicines Consortium (SMC) denied automatic access to the drug through the NHS in Scotland – which could see families compelled to move over the border in order to secure the drug for their sons.

In August 2014, Translarna became the first drug addressing a genetic cause of Duchenne muscular dystrophy to be approved in the EU. It has been available to families in a number of European countries for over a year.

The families of just over 50 eligible children in England – 60 across the UK – have waited almost 18 months for a decision from NHS England on funding for the drug.

The new agreement is intended to cover access to Translarna for eligible children in England for five years.

The news comes as a victory for families who have campaigned with Muscular Dystrophy UK. Boys delivered hand-written letters for the Prime Minister to 10 Downing Street.