Givinostat (distributed under the brand name Duvyzat) is a potential treatment for Duchenne muscular dystrophy (DMD). It received a conditional approval in the UK in December 2024. It is a drug from a family of molecules called HDAC (histone deacetylase) inhibitors that aims to reduce inflammation and fibrosis in muscle tissues, promote muscle regeneration and slow disease progression. It is manufactured by Italfarmaco and represented by ITF Pharma Ltd in the UK.
Givinostat
In March 2024 it was approved by the FDA (U.S. Food and Drug Administration) for use in boys aged six and older in the United States. In December 2024 the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) gave approval of a UK licence for Givinostat for use in both ambulatory and non-ambulatory patients aged six and over, on a conditional basis. We will update more about these conditions in early 2025 when the information is available.
Once licenced by the MHRA, a treatment must still be recommended by the National Institute for Health and Care Excellence (NICE) in order for NHS use to be possible in England, Wales and Northern Ireland; and by the Scottish Medicines Consortium (SMC) in Scotland.
The NICE appraisal process has already begun and we are working with other patient organisations on a joint submission to make sure that the community’s voice is shared with the decision-makers. A NICE Appraisal Committee meeting for givinostat is scheduled for May 2025. There is not currently a timetable for an SMC appraisal of givinostat.
Who is eligible for the EAP?
In order to be eligible to receive givinostat through the EAP, patients must meet a number of 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
Givinostat 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 the coming weeks, and we will keep you updated.
Accessing the EAP
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 givinostat 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.
Who should I speak to about accessing the EAP?
Please speak to your child’s Duchenne muscular dystrophy clinician/clinical team. Only Duchenne muscular dystrophy clinicians can make requests for givinostat for their patients. Decisions are made on a case-by-case basis for individual named patients aligned to eligibility criteria.
Working in partnership with Action Duchenne and Duchenne UK, we participated in the MHRA licensing process and will continue to work with them on the NICE and SMC appraisal processes.
Ahead of this, we hope that all NHS Trusts who receive a request from a North Star Centre to provide givinostat to an eligible patient approve this request. We will work with partners to ensure that Trusts understand the urgent need to make access possible for eligible patients.
Support and information
Duchenne muscular dystrophy is a muscle wasting condition caused by the lack of a protein called dystrophin. It usually affects only boys.
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.
We are here for everyone, but we understand that support isn’t one-size-fits-all. Speak to us. We can tailor our support to meet your unique needs.
Our helpline is open Monday-Thursday 10am-2pm.