Duchenne muscular dystrophy treatment givinostat conditionally approved in the UK

20 December 2024

Givinostat, a treatment for boys with Duchenne muscular dystrophy has been conditionally approved by the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK.

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 has now been approved in the UK for use in patients aged six years and older with all genetic changes associated with Duchenne muscular dystrophy (DMD).

Givinostat has been approved on a conditional license, meaning that further data is still needed before it will be granted a permanent license.

Next steps

Although the approval of givinostat in the UK is a welcome step forward, it is not yet available on the NHS. It must now 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). NICE and the SMC review how effective the treatment is and weigh it against the cost of the treatment. This process has started for givinostat, but a decision won’t be made until later in 2025.

In the meantime, to make sure boys with DMD can access givinostat, an Expanded Access Programme (EAP) has opened in the UK. This allows free-of-charge access for specific eligible patients while the process with NICE and SMC continues. Read more about the EAP.

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 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.