Duvyzat (also known as givinostat) is a potential treatment for Duchenne muscular dystrophy.
Duvyzat
Pharmaceutical company: ITF Pharma Ltd (Italfarmaco Group in the UK)
About the treatment
As our muscles are subjected to a lot of wear and tear, our cells have built in ways to help repair muscles and keep them functioning as they should. A molecule called histone deacetylase plays a key role in these repair processes. In people with Duchenne muscular dystrophy, histone deacetylase is overactive, reducing muscle repair.
Duvyzat blocks the activity of histone deacetylase, to help improve muscle repair. It aims to reduce inflammation and the build-up of scar tissues, and ultimately slow progression. Duvyzat comes as a liquid that can be swallowed.
Can I (or my child) get access now?
While the approval process is ongoing, people with Duchenne muscular dystrophy may be able to access givinostat through an Expanded Access Programme (EAP). This allows for free-of-charge access for specific eligible patients. Participation in the EAP for givinostat must be through one of the NorthStar Centres.
Current status
| Can it be prescribed in the UK? | Yes, but only if additional data is collected and reviewed |
| Is it available through the NHS? | England: No – assessment in progress |
| Wales: No – assessment in progress |
| Northern Ireland: No – assessment in progress |
| Scotland: No – assessment not started |
| Access to Duvyzat may be available through an Expanded Access Programme while the assessments are completed. |
In December 2024, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) approved Duvyzat for use in both ambulatory and non-ambulatory patients aged six and over, on a conditional basis.
As Duchenne muscular dystrophy has limited treatment options, a conditional approval means the treatment can be accessed while additional data is collected to confirm it works. The MHRA has said they require further data to confirm if Duvyzat works for patients who are not able to walk independently (non-ambulatory).
What happens next?
The National Institute for Health and Care Excellence (NICE) are reviewing the submission for Duvyzat, with a decision expected in 2025. If recommended, Duvyzat will be available in England, Wales and Northern Ireland.
There currently isn’t a timeline for the assessment (by the Scottish Medicines Consortium) for use on the NHS in Scotland.
Our involvement
We are working in partnership with the charities Action Duchenne and Duchenne UK to make sure that the community’s voice is heard during the NICE assessment.
While the assessment is ongoing, we will work with partners to ensure that NHS Trusts understand the urgent need to make access to Duvyzat possible for eligible patients through the EAP. We hope that all NHS Trusts who receive a request from a NorthStar Centre to provide Duvyzat to an eligible patient approve the request.
Clinical trial results
The phase 3 EPIDYS clinical trial included 179 boys with DMD, aged six and older and could walk. They were randomly assigned to receive Duvyzat (118 participants) or placebo (dummy drug, 61 participants).
After 72 weeks, all participants’ four-stair climb assessment results got worse, suggesting their ability to move reduced. However, the decline in movement was statistically significantly smaller in those who received Duvyzat compared to those who received the placebo. This suggests Duvyzat is likely to slow progression. Duvyzat was found to be safe. Some participants had a reduction in blood platelets (which help with clotting and to stop bleeding), but this was managed by reducing the dose.
Last updated: 18/02/2025
Frequently Asked Questions on accessing Duvyzat
To be eligible to receive Duvyzat through the EAP, patients must meet several criteria.
These include:
- A confirmed diagnosis of Duchenne muscular dystrophy
- Age six and above, and ambulant (able to walk)
- Residing lawfully in the UK on a properly settled basis
Duvvzat has also been approved on a conditional license for patients aged six years and older, who are not able to walk independently (non-ambulant). This means that further data is still needed before it will be granted a permanent license. The company have said they are committed to gather this additional data through an EAP. More details on this EAP will be announced in early 2025, and we will keep you updated.
The EAP can only be administered through specialist sites, which means the 23 paediatric neuromuscular centres across the UK that are part of the NorthStar network.
However, participation in the EAP is decided at an individual NHS Trust level and a North Star Centre will not be able to provide Duvyzat if its local Trust has not approved participation. A request to a Trust to approve participation in the EAP is generally led by the Duchenne muscular dystrophy clinical lead. This is generally via the Trust’s Drugs & Therapeutics (D&T) committee.
Please speak to your child’s Duchenne muscular dystrophy clinician/clinical team. Only Duchenne muscular dystrophy clinicians can make requests for Duvyzat for their patients. Decisions are made on a case-by-case basis for individual named patients aligned to eligibility criteria.
Support and information
Duchenne muscular dystrophy
Duchenne muscular dystrophy is a muscle wasting condition caused by the lack of a protein called dystrophin. It usually affects only boys.
Research
We fund groundbreaking research to learn more about muscle wasting conditions and lead us to new treatments. We’ve already made advances that would have been unthinkable just 10 years ago, and we are determined to go even further and faster.
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