Promising drug on its way

Trials run with Duchenne UK families show Givinostat can slow Duchenne muscular dystrophy (DMD) progression in boys aged six and older

Promising results

Our global reach means that our DMD Hub is involved in the world’s leading drug trials, all focused on finding treatments for DMD patients as soon as possible.

Our DMD Hub recruited patients in the UK for a worldwide trial of Givinostat.

The trial was delivered at four of the eleven DMD Hub sites; Newcastle, Alder Hey, Oswestry and Great Ormond Street Hospital.

The phase three evaluation of Givinostat demonstrated that it can slow DMD progression in boys aged six and up. Givinostat is a histone deacetylase (HDAC) inhibitor that is produced by the Italian pharmaceutical company Italfarmaco.

Getting treatment to patients

The European Medicine Agency (EMA) is now beginning its approval process, and US regulators are doing the same.

This is good news for patients in the UK. If Givinostat is approved by the EMA, its producer can use this approval to seek a quicker approval by the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK.

We aim to work with Italfarmaco to support the next steps towards UK regulatory approvals to ensure that everyone living with DMD who could benefit from this treatment has access to it.

Duchenne UK Impact Report 2023

A landmark year of achievement and impact