Givinostat (Duvyzat®) has had conditional European marketing authorisation since June 2025 for boys aged 6 years and older with Duchenne muscular dystrophy (DMD) who are ambulatory and also treated with corticosteroids. On 12 February 2026, the French National Authority for Health (HAS) issued an unfavourable opinion on its reimbursement by the national health insurance system for this indication, halting the process that would have allowed the drug to be marketed in France.
The HAS concluded that givinostat (Duvyzat®) provided insufficient medical benefit (SMRi) for this indication, based on:
- insignificant efficacy results, particularly for the primary endpoint: the time taken to climb four steps decreased by only 1.78 seconds on average in patients treated with givinostat for 18 months compared to the placebo group in the Epidys trial;
- a difference in the time taken to get up from the floor (first secondary endpoint) that is not significant between the two groups;
- significant haematological and gastrointestinal side effects.
French patients who received the drug under a compassionate use programme will continue to be treated for at least one year, in accordance with the law.
Further laboratory results are expected by 2033 as part of a new Phase III trial, as required by the European Agency in relation to conditional marketing authorisation.
