Duchenne drug Translarna gets the green light

Published Date
Alexa Follen

After months of campaigning, driven by the support of families, Muscular Dystrophy UK is thrilled to bring you the final ‘yes’! NHS England and PTC Therapeutics have reached an agreement, and NICE has signed off its final guidance: NHS England will fund Duchenne muscular dystrophy drug, Translarna.

This is history in the making: Translarna is the first-ever drug to target an underlying genetic cause of a form of muscular dystrophy. The treatment is the first ever drug to target an underlying genetic cause of a form of muscular dystrophy. Under the terms of the funding agreement, it would be used to treat children whose condition is caused by a nonsense mutation, who are aged five and over and who are able to walk.

What happens now?

This is the last stage of the process: the drug has now finally been approved for funding on the NHS in England. As part of the agreement, NHS England has waived the usual 3-month funding period, meaning the drug could be available within weeks.

Not sure if your child is eligible? You should speak to your specialist and we have a factsheet on our website.

Information for health professionals

NICE have now signed off final guidance on Translarna, the first-ever drug to target an underlying genetic cause of a form of muscular dystrophy. Muscular Dystrophy UK has been in touch with NHS England, who have advised that clinicians will now need to initiate appointments to conduct baseline assessments for patients going onto the Managed Access Agreement. In addition, it will be necessary to approach your centre’s hospital pharmacy team, who will then need to liaise with the local NHS England pharmacist to ensure that the appropriate steps are taken to make the drug available in clinic. We understand more detailed guidance for specialist teams will be issued by NHS England shortly. In the meantime, please do get in touch if you would like any further information.

What about the rest of the UK?

We expect this decision to lead to implementation in Wales and Northern Ireland and we are working closely with Health Ministers and their officials to ensure that the required preparatory work is undertaken to result in availability of Translarna as soon as possible.

In Scotland, the process has been entirely separate. The Scottish Medicines Consortium has rejected the drug for overall approval, and the campaign is well underway to secure NHS funding.

In the Isle of Man, the drug has also not been approved for funding. Muscular Dystrophy UK is working with the family affected to put pressure on the Isle of Man government to reverse its decision.

Sue Barnley, whose 7-year-old son, Harry, has Duchenne muscular dystrophy, said: 

As a family living with Duchenne, you will understand just how much this means to us. After two years of uncertainty, Harry will finally be able to receive Translarna on the NHS. Thanks to every one of you who helped to make this happen. Giving our beautiful and cheeky boy extra time to play like other children his age is truly priceless.”

Robert Meadowcroft, Chief Executive of Muscular Dystrophy UK, said: 

Families in England will deservedly be thrilled by today’s news from NICE. Together, we have fought long and hard for this pioneering drug to be made available on the NHS. For all those who campaigned with such determination, today is a very welcome relief.


Duchenne is a devastating condition and it’s almost too much to bear for parents to watch their child’s health worsen day by day as it progresses. By keeping these boys mobile, Translarna could also enable them to be eligible for other future drugs on the horizon.


While NICE’s announcement is welcome, it is simply unacceptable that Scotland is forced to continue fighting, with a cruel postcode lottery for families to endure. We will not rest until all eligible boys can access this drug.”

 Sir Andrew Dillon, Chief Executive at NICE, said: 

Duchenne muscular dystrophy caused by a nonsense mutation is a cruel disease that currently has few treatment options. Ataluren is an innovative drug that for the first time is aimed at the root cause of the disease and has the potential to offer benefits to people with the condition and their families.


When we published our draft recommendations in April we acknowledged that ataluren represents a significant cost to the NHS at a time of increased pressure on funding, especially given the uncertainties of the drug’s potential long-term benefits.


“We are therefore extremely pleased NHS England and the company have agreed the terms of a 5-year managed access agreement for ataluren. The agreement means children with this condition will now be able to access the drug while at the same time allowing more data to be gathered on its efficacy, before the guidance is reviewed and a further decision made on whether funding should be continued after 5 years.

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

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