Givinostat: more evidence needed before a decision on NHS access in England
11 August 2025
Givinostat, a potential treatment for Duchenne muscular dystrophy, is being reviewed to see if it should be made available on the NHS in England. A decision hasn’t been made yet, as the committee has asked for more evidence to help them decide. This has caused a delay, which we’re disappointed by, and adds to the challenges families face in accessing treatment.
In clinical trials, givinostat has helped slow down how quickly Duchenne muscular dystrophy (DMD) gets worse. This could make a big difference to children and families living with the condition.
Right now, givinostat is being reviewed by an expert group from NICE (National Institute of Health and Care Excellence). They help decide which treatments should be available on the NHS in England. After their first meeting on 10 July 2025, they have now asked for more evidence before making a decision. While we welcome the need for strong evidence, we’re disappointed by this delay. It adds to the challenges families currently face in accessing treatment.
How does the review work?
Before a treatment can be offered on the NHS, it goes through a careful review. This looks at:
How well the treatment works and how safe it is
Whether it’s good value for money
The impact of living with the condition
To help with this, NICE looks at evidence from the company that makes the treatment, doctors and patient organisations like ours. We share what it’s like to live with the condition, what matters most to families and how the treatment has made a difference to their lives. People affected by DMD can also speak at the meeting to share their experiences directly.
The evidence includes data from clinical trials and calculations that try to show how much the treatment helps, how long the benefits last, and whether the cost is worth it. An independent team checks this information to make sure it’s fair. If needed, they suggest different ways to look at the data to help the committee make the best decision.
What did NICE say about givinostat?
Usually, after this first meeting that happened on 10 July 2025, NICE either:
Recommends the treatment for NHS use, or
Doesn’t recommend it but asks for more discussion.
This time, the committee took a different route. They’ve asked for more evidence before they can decide. This means they haven’t said yes or no yet.
The committee didn’t agree with some of the calculations used to show if the treatment offers good value for money. These calculations came from both the company and the independent team. NICE now wants more data to help them understand the benefits of givinostat and whether it should be funded by the NHS.
Why does this matter?
Some families are getting givinostat through an expanded access programme (EAP), where the company provides the treatment for free while the review is still happening. But this programme isn’t available everywhere in the UK, meaning some families are missing out on a potentially life-changing treatment. Find out more about the EAP in our previous blog.
The EAP was designed to support access while a decision is being made, but it’s not reaching everyone who could benefit. While over 100 boys are currently receiving givinostat through the EAP, many more could benefit. Given this new delay, access to the EAP must improve so that more boys can get the treatment.
Every month without a decision means more waiting. And for a progressive condition like DMD, time really matters. Families affected by DMD deserve equal access to promising treatments, no matter where they live.
What happens next?
The company has one month to send in more evidence.
The independent team will check it.
The committee will meet again in October.
We know this delay is frustrating and we have shared our concerns directly with NICE. We’ll keep speaking up for everyone living with muscle wasting conditions and share updates as soon as we know more.
“We’re disappointed by the delay in the NHS England decision on givinostat and have shared our concerns with NICE. Families affected by Duchenne muscular dystrophy are already facing unequal access to this treatment through the early access programme, which isn’t available everywhere in the UK. Every month of delay adds to the uncertainty and means more families missing out. This is yet another example of how the system isn’t working for people with muscle wasting conditions. We urge NICE and the company to act swiftly and collaboratively, and for more centres to join the EAP, so families across the UK can access the care they deserve.”
