A 4-year-old boy from East Lothian has become the first child in the UK with Duchenne muscular dystrophy to receive NHS funding for Translarna.
Cormac Fegan will begin receiving the drug Translarna for at least six months after he reaches his fifth birthday in November.
It is the first drug available in the EU to treat an underlying cause of the life-limiting muscle-wasting condition, and could keep Cormac walking for longer. Health professionals confirmed to Cormac’s parents they would gain access to Translarna, following the approval of an Individual Funding Request.
Translarna has been available in several European countries since it was approved by the EU in August last year.
The NHS refused to fund the drug, meaning parents may have to wait until next year before a decision is made by NICE, which is expected to release its draft guidance for the NHS on whether it should fund the drug within weeks.
NICE’s decision has a direct impact on whether the treatment is made available in Northern Ireland and Wales. Individual Funding Requests made by families in Northern Ireland and England have previously been rejected or put on hold.
The drug could delay the need for children to use a wheelchair. To be eligible for Translarna, children must still be able to walk. It is feared delays could mean some miss out on the chance to benefit from Translarna entirely.
This breaking story has been covered across Scotland and the rest of the UK, including BBC Online, the Daily Mirror, the Daily Record, Herald Scotland and a live interview on BBC Radio Scotland (48.40)
We, along with other patient organisations, are pressing for NICE to reach a positive decision on access to Translarna so that boys who are eligible for the treatment are able to access the treatment across the UK.
Gary Fegan, Cormac’s father, said:
This is really exciting for us – we feel very lucky that the timings have worked out, as Cormac is about to have his fifth birthday, the age when you can start taking Translarna. This gives us hope for the future and when new and innovative drugs come through in Scotland, Cormac should be eligible.
We’ve heard all the horror stories from people in other parts of the UK, so we were prepared to have a fight on our hands. We felt a huge sense of relief when we received the news. We really hope the decision from NHS Lothian will put more pressure on NICE and the NHS to provide Translarna for all boys who are eligible.
Sue Barnley from Hampshire, whose son Harry has Duchenne muscular dystrophy and is eligible for the Translarna, said:
Living with Duchenne is living with a black cloud, made worse by the constant delays in decision-making. As a family, waiting for this decision has been agonising. We need NICE and the NHS to understand that we don’t have the luxury of time. Every day matters, every day these boys are losing mobility.
I am thankful to the NHS in Scotland for giving Cormac and his family some real hope, it is absolutely the right decision, and one that we hope will encourage the decision-makers in England to follow suit. We need them to give approval now and get this drug out to all the boys who will benefit without delay.
Robert Meadowcroft, Chief Executive of Muscular Dystrophy UK, said:
We are encouraged by this decision. This is the first time a therapy addressing a genetic cause of Duchenne muscular dystrophy will be funded by the NHS. We hope that Cormac’s case will be used as a benchmark for all the families in Scotland, and indeed the rest of the UK.
The true value of being able to walk for longer must not be underestimated. Duchenne is a life-limiting condition, and we must take every opportunity to protect children and their quality of life. It is vital that NICE and the NHS recognise that all eligible boys must be able to access this drug as a matter of urgency. We may not yet be able to halt the difficult challenges these children face, but with this innovative drug, we have the opportunity to delay them.
It is unacceptable that a family’s location in the UK will dictate whether their child receives this breakthrough drug or not.
What can I and my family do to help?
We’re asking our MP supporters to urgently contact NICE and call for a positive decision on Translarna.
We need your help to do this: we’re calling on all families affected by Duchenne to contact their MP, asking them to write to the Chief Executive of NICE, Sir Andrew Dillon.
Ask your friends, families, and neighbours to do the same using our template letter.
For more information on the Translarna campaign, please contact Peter Sutton on firstname.lastname@example.org