The decision on whether Duchenne muscular dystrophy treatment, givinostat, can be offered on the NHS in England for patients aged 6 years and older has been on pause since late 2025, and that pause is now continuing. We know this delay is frustrating, but there are encouraging signs: final discussions are progressing, and the review is now waiting for new rules in April that could help more treatments be approved.
Givinostat has been looked at by the National Institute for Health and Care Excellence (NICE) for more than a year. NICE decides which treatments can be used on the NHS in England, and its decisions are usually followed in Wales and Northern Ireland. Although givinostat was approved for NHS use in Scotland in December 2025, families in the rest of the UK are still waiting for NICE’s final decision.
Why the delay?
In late 2025, NICE announced they had paused the review to allow the company who makes givinostat, ITF Pharma UK, and NHS England to negotiate the price of the treatment. These discussions are still ongoing and need more time, which is why the pause remains in place.
We know this is incredibly frustrating and means continued uncertainty about long term access.
Final discussions between ITF Pharma UK and NHS England are ongoing, and the pause gives the time needed for these conversations to reach a conclusion. It also allows the review to restart under updated NICE rules coming in April, which are designed to give more treatments a chance of being approved.
Taken together, these developments mean that givinostat is in a stronger position than it was a few months ago. Although we cannot predict the final decision, we are hopeful that these changes will support a positive outcome.
What are the new NICE rules?
NICE is updating the way it judges whether treatments offer good value for the NHS. This includes increasing the threshold it uses when deciding if a treatment provides enough benefit for its cost. The change is expected to help more treatments be approved than before, especially those that were very close to meeting the previous criteria.
The new rules are expected to come into effect in April, once they are approved by government. However, there is still some uncertainty about exact timelines. This means we don’t yet know when the review will restart or when the next update will be available. We will let you know as soon as we hear more.
What this means for families right now
The Expanded Access Programme is expected to continue. While some families can get the treatment through an EAP, others still cannot. The delay means access to givinostat remains uneven across the country
The EAP allows certain boys and young men to receive givinostat before a full NHS decision is made, and ITF Pharma UK has been supplying the treatment to the NHS free of charge through this programme.
However, not all centres have been able to take part because the EAP does not fund the staff time or resources needed to deliver the treatment safely. These centres have said they need to wait until NICE’s final decision and has meant that some who are eligible have so far been unable to access it.
We understand that this is frustrating and together with the charities Duchenne UK and Muscular Dystrophy UK we have been pushing ITF Pharma UK, NHS England and NICE throughout this pause to reach a solution as quickly as possible – one that allows givinostat to move into routine NHS use so that those who can benefit can access it.
We will keep pushing urgently until this becomes a reality.
Our Support:
We are aware that further delay on this decision is incredibly frustrating and want to remind you that we are here to support you and your family wherever you are on your journey. If you would like to talk to a member of our Support Team, contact us on 07535498506 or use the form on our website. We also have monthly online support groups for Duchenne mums, dads and grandparents. Our next groups will be held in the first week of April and you can find out how to join us here.
To make sure you stay up to date on all developments as well as receiving more details of our support services, sign up to our monthly newsletter.
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