The fight for access to Translarna

Translarna is the first ever drug to potentially treat Duchenne muscular dystrophy, and one of the first for any muscle-wasting condition, to be approved outside of clinical trial.

The drug was granted conditional approval by the European Commission in August 2014. It is designed to treat boys whose Duchenne is caused by a nonsense mutation, who are aged five and over and who can still walk.

Translarna is already available in countries including France, Italy, Spain, Germany and Denmark, but it is not yet approved here in the UK.

Why is fast access so important?

Clinical trials of Translarna show that the drug would help keep children walking for longer, and delay the need for a wheelchair. It could also delay the later devastating stages of the condition, including breathing difficulties.

However, children must still be able to walk in order to obtain the treatment, so months of delays could be the difference between some being able to access the drug or not.

Once lost, muscle fibres cannot be restored. Every day counts.

I know that I won’t always be able to walk, but I would like to for as long as I can. Translarna is helping me to do that. Please help me to keep taking the drug, and make sure that other boys who it could be helping have it as soon as possible

11 year old George Pegg, is on a clinical trial for Translarna. He went with five other children affected by Duchenne, to deliver handwritten letters to the Prime Minister, asking for access to Translarna.

Fighting for access

With families, we are fighting for access to the drug. We organised for families and the boys to take a massive petition to Downing Street and we have taken the campaign directly to decision makers –NHS England, senior Health Ministers, NICE and even a meeting with the Prime Minister.

He could live a longer, fuller, better quality life. The impact on us as parents and his wider family would be immense. We wouldn’t have to go through the helpless watching as Duchenne takes over…It would slow this process down. Harry’s sister Maisie is just 21 months older than he is. They are very close. She is looking forward to him starting school, so she can help him, as that’s what big sisters do. It will hit her very hard when Harry is no longer able to walk.

Helen Duell, whose son, Harry, is eligible for Translarna

What next?

In July 2015, NHS England further delayed a decision on Translarna, saying that they would wait for a recommendation from NICE.

In October 2015, NICE announced that it is ‘minded not to approve’ Translarna, instead asking for more information from the drug company on clinical trial and the costs of the treatment.

The announcement from NICE is not a final decision, and Muscular Dystrophy UK, the drug company and fellow charities have submitted further evidence. You can read our response here.

NICE met again on 17 November, and a recommendation on final guidance is likely to be made this year.

We’ll keep pulling out all the stops for a positive decision.

For more information, please contact Peter Sutton on p.sutton@musculardystrophyuk.org or call 020 7803 4838.

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