The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending the conditional marketing authorization of givinostat for the treatment of ambulant patients aged six years and older with Duchenne muscular dystrophy (DMD).

The European Commission is expected to make a final decision on the authorization of the medicine within the European Union in July.

Duchenne muscular dystrophy is a rare, progressive neuromuscular disorder caused by mutations in the DMD gene, which make muscle fibers more vulnerable to damage and prevent the activation of key genes essential for muscle maintenance and repair. The continuous cycle of muscle injury leads to chronic inflammation, impaired regeneration, and the gradual replacement of muscle tissue with connective and fatty tissue. The condition primarily affects boys, with symptoms typically appearing between the ages of two and five. Over time, muscle weakness worsens, affecting mobility and eventually involving the heart and respiratory muscles, often leading to premature death. DMD is among the most common types of childhood muscular dystrophy, with a global incidence of approximately 1 in 5,050 boys.

For more information, please contact Tina Vučković, President of DMD Hrvatska (Duchenne Muscular Dystrophy Association of Croatia), by phone at +385 91 9138 010 or via email at dmdhrvatska@gmail.com. dmdhrvatska@gmail.com.