Update from NICE about givinostat appraisal
The National Institute for Health and Care Excellence’s (NICE) appraisal of givinostat (Duvyzat), to decide whether the medicine will be approved for treatment of Duchenne muscular dystrophy (DMD) on the NHS in England, has now been ongoing for more than a year, and we know many families have been anxiously waiting for news.
NICE has just shared the following update
“This appraisal continues to be paused to allow the company and NHS England to finalise commercial discussions and until NICE has the power to apply the new cost-effectiveness threshold.
“NICE anticipates that its cost-effectiveness threshold will increase in April 2026 and we will provide a further update to stakeholders in due course. More information about changes to NICE’s cost-effectiveness thresholds can be found here.”
You can read the statement on the NICE website.
We know this update will be frustrating for families, especially for those still unable to access givinostat, as it means further delays and ongoing uncertainty.
What does this mean?
In December 2025, NICE announced it had paused its review of givinostat to allow the company that makes it, ITF Pharma UK, and NHS England, to enter into commercial negotiations.
The pause is now continuing to give the parties time to finalise these commercial discussions. It will also allow NICE to apply changes the government is introducing to the thresholds it uses to judge whether a medicine is cost-effective for use on the NHS, which could improve givinostat’s chances of approval.
We cannot predict NICE’s final decision but hope these new developments will support a positive outcome.
What are the new NICE cost-effectiveness thresholds?
The cost-effectiveness thresholds are the measure NICE uses to judge whether a medicine delivers enough health benefit for its cost and whether it should be approved for use on the NHS.
The government announced last year that it would seek to increase the cost-effectiveness threshold from £20,000 to £30,000, to £25,000 to £35,000, with the changes due to be introduced in April 2026.
The increase could enable NICE to approve some medicines that previously would not have been considered cost-effective.
NICE cannot implement the new measure until it receives a direction from the Secretary of State for Health and Social Care. This requires some changes to NICE regulations, made through legislation, known as statutory instruments. Following a consultation, which concluded earlier this year, the legislation has now been laid before parliament, and the changes should be on track to be introduced in April.
When will we know more?
NICE has not provided a timeline for resuming the appraisal or for further updates. All we know is that it will be in or after April, once the new NICE thresholds are in place.
As stakeholders, representing the patient community, alongside Muscular Dystrophy UK and Action Duchenne, we continue to engage with NICE, NHS England and ITF Pharma, to push them to reach agreement and to bring this appraisal to a positive conclusion, as quickly as possible.
As soon as we have further news, we will provide an update.
What does this mean for families now?
Givinostat was given marketing authorisation by the Medicines and Healthcare Products Regulatory Agency (MHRA) in December 2024, deeming it safe and effective. It has now been looked at by NICE for more than a year to determine whether it can be used routinely for all eligible patients on the NHS in England.
Some families have already been able to access givinostat via an Early Access Programme (EAP). Through the EAP, ITF Pharma UK is providing the medicine free-of-charge to participating NHS Trusts, who are making it available to eligible ambulant patients.
Families already receiving the medicine through the EAP will continue to do so. The EAP should also remain open to new enrolments, until NICE issues guidance on givinostat.
Unfortunately, not all NHS Trusts treating patients with DMD are taking part in the EAP, meaning eligible patients in some areas are still being denied access to this medicine.
There is also still no access for non-ambulant patients. We continue to explore ways to make this possible.
Postcode lottery
Givinostat has been being looked at by NICE for more than a year to determine whether it can be used routinely for all eligible patients on the NHS in England. NICE decisions are usually also followed in Wales and Northern Ireland.
Meanwhile, givinostat is already routinely available in Scotland for ambulant patients who are six years old and over, after it was accepted for use on NHS Scotland by the Scottish Medicines Consortium (SMC) in December 2025.
This postcode lottery of access is extremely unfair, and we know how devastating it is for those families still unable to access this medicine.
We continue to highlight the inequity and to call on decision-makers to reach a final decision, as swiftly as possible, so all eligible patients can benefit from this treatment.