On the 25th of April, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) gave Givinostat (Duvyzat) a positive opinion. The recommendation is for granting a conditional marketing authorisation for Givinostat for the treatment of ambulant patients with Duchenne muscular dystrophy (DMD) aged six years, and alongside concomitant corticosteroid treatment in the European Union (EU).
This positive recommendation needs to be reviewed by the European Commission (EC) within 67 days before the treatment is made available in the EU. The European Commission will review the CHMP’s recommendation and is expected to make a final decision in July 2025.
Notable differences between MHRA (UK) and CHMP (EU) indications
In the EU, the proposed indication for Givinostat is for ambulant patients only, and to be utilised alongside corticosteroid treatment. In the UK, the Medicines and Healthcare products Regulatory Agency (MHRA) issued a market authorisation that applies to both ambulant and non-ambulant patients. Furthermore, in the UK, there is no specific additional requirement for Givinostat treated patients to also be on corticosteroid treatment.
In the UK, Givinostat is currently being assessed by the National Institute for Health and Care Excellence (NICE) for routine access via the NHS. NICE’s committee meeting is scheduled for July 2025, with a provisional date for a decision in September 2025.
Givinostat (Duvyzat)
Givinostat is an investigational drug for Duchenne muscular dystrophy (DMD) that belongs to a class of medications called histone deacetylase (HDAC) inhibitors. The aim of the treatment is to reduce inflammation and fibrosis (scarring) in muscle tissue, and potentially promote muscle regeneration, thereby aiming to slow down the progression of the disease.
Givinostat Early Access Program (EAP):
Givinostat has yet to receive a decision by the NICE to make the treatment available under the NHS. However, the treatment manufacturer (ITF Group) has made givinostat freely available through NHS clinics for eligible patients, via a compassionate access scheme termed an Early Access Program.
