Breaking News: First clinic doses patients with givinostat under Early Access Programme
Duchenne UK is delighted to report a landmark achievement: the first boy with Duchenne muscular dystrophy (DMD) in the UK has received givinostat through the Early Access Programme (EAP). This is the first time a person with DMD in the UK has been able to access the medicine without participating in a clinical trial.
Whilst this is a moment for celebration, Duchenne UK is acutely aware that hundreds of families across the UK are desperately hoping for their sons to be given givinostat under the EAP too.
This is why our Time is Muscle campaign has been fighting so hard since November. And it does not stop here. Duchenne UK will not stop campaigning until either every DMD clinic is taking part in the EAP, or until NICE gives a positive opinion to givinostat and it is made available on the NHS.
Eligible patients at the Leicester Royal Infirmary will receive their first dose of givinostat through the EAP this month. Charlotte Wakeling’s 10-year-old son Felix had his first dose this morning. She said,
“I started writing to my local NHS trust, the MHRA and my MP in December 2024 after Duchenne UK first shared news of the EAP. The CEO of our trust has supported Dr Dhinesh Baskaran and his team in implementing the EAP and they have worked really hard to get it in place, as quickly as possible, for all eligible boys. I am delighted that it has finally happened.
“Now that Leicester Royal Infirmary has enabled access to givinostat, I urge the rest of the NHS to follow their example and provide access to this free treatment to all eligible patients as quickly as possible.”
Charlotte worked with her constituency MP to enable access to givinostat. Dr Luke Evans, Member of Parliament for Hinckley and Bosworth, said,
“I have seen first hand the devastating impact that muscular dystrophy can have, not only on the patient but the family as a whole. So, to now have the chance as an MP to help patients get access to a drug that can make a difference was an opportunity I felt strongly about.
“I’m grateful to the team at University Hospitals of Leicester for working with Charlotte and I to secure treatment for Felix and patients across our area. It’s a great step forward. I hope to see other NHS Trusts striving to take part in the givinostat Early Access Programme soon.”
Alex Johnson OBE, founder of Duchenne UK and Joining Jack, said,
“Our families are doing everything they possible can to get their sons to take part in the givinostat EAP, and whilst we are thrilled at this news, we are still desperately anxious for those families who can’t access the drug, a drug that remember is being offered FREE to the NHS.”
Duchenne UK’s co-founder and CEO, Emily Reuben OBE, said,
“If a medicine for a devastating disease like DMD works, then everyone who could benefit deserves access. This has been a long time coming, but we are grateful that the team at Leicester Royal Infirmary has put in the work behind the scenes to facilitate access to the EAP for their patients.
“We now call on the other NHS Trusts and Boards which host specialist DMD clinics to follow Leicester Royal Infirmary’s example, and take part in the givinostat EAP. Time is muscle, and we can’t afford a delay.”
Givinostat is a treatment for DMD which was licensed in the US in March 2024, and in the UK in December 2024 for all people living with DMD age six and above. It is not a cure, but it’s been proved to slow the progression of the disease in clinical trials. While the treatment is being assessed for cost-effectiveness in the NHS, it is being made available for free to the NHS by the manufacturer through the EAP – a type of compassionate use scheme.
Duchenne UK has been campaigning for urgent access to givinostat through the EAP with our Time is Muscle campaign since we first shared news of it in November 2024. Along with Muscular Dystrophy UK, we then wrote to the CEOs of all 24 NHS trusts in the UK neuromuscular expert NorthStar network, making them aware of the EAP, offering to help, and urging them to take part. On 19 December, we held a community webinar with clinicians from the NorthStar network so further information on givinostat and the EAP could be shared.
And the community response to the news has been immense.
Over 200 letters on givinostat were sent to the Medicines and Healthcare products Regulatory Agency (MHRA), which helped result in a broad label licence in December for everyone with DMD over 6 years old, regardless of ambulation (walking) status. Dozens wrote to their local NHS trust and their MP about the EAP. And on 22 January we met with 30 MPs in Parliament – including Health Secretary Wes Streeting – to discuss how important access to givinostat is.
Uptake of this free and approved medicine through the EAP has been slow, but now that Leicester Royal Infirmary has led the way, we call on other NHS trusts to follow their example and take part in the EAP. If Leicester Royal Infirmary has been able to solve their challenges, then other NHS trusts can too. The Time is Muscle campaign does not stop here. Duchenne UK will carry on campaigning on givinostat until it’s available for everyone with DMD who could benefit.
What is givinostat?
Givinostat is not a cure for DMD, though it may help preserve muscle function and slow down disease progression. It is an ‘HDAC inhibitor’. It blocks enzymes called histone deacetylases (HDACs), which are involved in turning genes ‘on’ and ‘off’ within cells. It works by targeting pathogenic processes to reduce inflammation and muscle loss. You can read a more technical explanation of givinostat by Professor Annemieke Aartsma-Rus on the World Duchenne Organisation’s website here.
We will be discussing the latest development on givinostat and the EAP at our New Horizons conference that’s taking place in London from Friday 28 – Saturday 29 March 2025. You can find out more about it and register for a place here.
Givinostat will be assessed by the National Institute for Health and Care Excellence (NICE) for routine use in the NHS in May.