An Early Access Programme (EAP) is being made available to eligible people with Duchenne muscular dystrophy to access a new treatment called givinostat.
Early Access Programme for new Duchenne muscular dystrophy treatment givinostat
15 November 2024
Givinostat 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 not currently licensed in the UK. It is manufactured by Italfarmaco, and represented by ITF Pharma Ltd in the UK.
Earlier this year it was approved by the FDA (U.S. Food and Drug Administration) for use in boys aged six and older in the United States. Although not currently licensed in the UK, the process for getting a UK licence is underway. It must then be recommended for use in England, Wales and Northern Ireland by the National Institute for Health and Care Excellence (NICE); and in Scotland by the Scottish Medicines Consortium (SMC).
Access for eligible patients in the UK may be possible now though, thanks to an Expanded Access Programme (EAP) opened by ITF Pharma UK. This allows free-of-charge access for specific eligible patients until approvals are in place.
The aim of the givinostat EAP is to provide access to givinostat for eligible patients with DMD because of the lack of other treatments being available.
There are a number of criteria that people need to meet in order to be eligible for the EAP. Further detail about these criteria and more information about givinostat and the EAP are available.
Access to the EAP must be through one of the 23 NorthStar Centres in the UK. 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. Under the EAP, givinostat is free to both patients taking part in it and to the NHS, but the NHS Trusts must still cover the cost of administering it to patients.
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. If you think your child might be eligible for the EAP or have questions about it, please speak to their Duchenne muscular dystrophy clinician/clinical team.
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 patient.
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