Access to treatments

Givinostat recommended for approval in the EU

Today, 25 April 2025, a committee of the European Medicines Agency (EMA) recommended conditional authorisation for givinostat for the treatment of Duchenne muscular dystrophy (DMD) in ambulant patients, aged 6 years and older, and with concomitant corticosteroid treatment. Full approval requires acceptance by the European Commission (EC). This announcement does not affect patients in the UK, but it will be welcome news for patients in Europe.

The EMA’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion for givinostat, recommending it for ambulant boys over the age of 6, when treated alongside corticosteroids. This is different to the approval from the UK’s Medicines and Healthcare Regulatory Agency (MHRA), which did not add the requirements on ambulation (walking) and corticosteroids.

The CHMP’s recommendation will not become final until it is accepted by the EC. The EC aims to respond to the recommendation within 67 days. If accepted by the EC, the centralised marketing authorisation is valid in all EU Member States as well as in the European Economic Area countries Iceland, Liechtenstein and Norway.

Decisions by the CHMP and the EC do not change anything for British patients. In the UK, givinostat has market authorisation from the MHRA and is currently being assessed by the National Institute for Health and Care Excellence (NICE) for routine access in the NHS. NICE’s committee meeting is scheduled for July, with a provisional date for a decision in September.

While it is being assessed by NICE, givinostat is available for free to NHS clinics through a compassionate use scheme called the Early Access Programme (EAP). Duchenne UK has been campaigning for all eligible clinics to take part in the EAP through our Time is Muscle campaign.

For more information on how drugs gain market authorisation in the European Union, click here.