The European Commission has granted conditional marketing authorisation for givinostat (marketed as Duvyzat), a novel treatment for Duchenne muscular dystrophy (DMD) – a rare and fatal genetic disease causing progressive muscle degeneration in children. This decision follows a positive scientific opinion from the European Medicines Agency (EMA), which concluded that the immediate availability of the treatment outweighs the risks linked to incomplete data.

Duvyzat, an oral suspension used alongside corticosteroids, is approved for ambulant patients aged six and above. It works by inhibiting HDAC enzymes, which are linked to muscle damage and inflammation in DMD. In clinical trials, patients treated with givinostat showed a significantly slower decline in motor function than those on placebo.

Although promising, the authorisation is conditional and valid for one year, pending the completion of further studies to confirm long-term efficacy and safety. The most common side effects reported were mild to moderate and included digestive issues and changes in blood parameters.

This conditional approval marks a milestone in addressing an unmet medical need and could significantly improve quality of life for patients and families living with DMD – if ongoing studies confirm the initial findings.