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Europe’s Committee for Medicinal Products for Human Use (CHMP) has recommended granting a conditional marketing authorization for Duvyzat (givinostat, Italfarmaco) to treat Duchenne muscular dystrophy (DMD) in patients aged 6 years and older who can walk.

Conditional marketing authorization is granted when the benefit of immediate availability outweighs the risk of less comprehensive data than normally required. The marketing authorization holder is to provide extra clinical data at a later stage.

Duvyzat previously received orphan designation on July 4, 2012, for the same condition. The European Medicines Agency will now assess whether this designation should be maintained.

DMD is a rare muscle disorder characterized by progressive weakness and atrophy of the pelvic area followed by the shoulder muscles and other muscles of the body. It is typically recognized between 3 and 6 years of age and has a median life expectancy of 22 years.

The condition affects around 1 in 3500 newborn boys. Approximately 26,000 people have the condition in Europe. There is currently no cure for DMD, and treatment revolves around corticosteroid therapy, prevention of contractures, and care of the heart and respiratory function.

The active substance in Duvyzat is givinostat, a class I and II histone deacetylase (HDAC) inhibitor. It modulates uncontrolled activity of HDAC in dystrophic muscles, which reduces muscle fiber damage, chronic muscular inflammation, fibrosis, and fat deposition while promoting new mitochondria production. The drug’s precise mechanism of action in patients with DMD remains unknown.

The CHMP’s recommendation is based on data from a subgroup of 120 patients with ambulant DMD who took part in a randomized, placebo-controlled study. Patients were aged 6 years and older and had concomitant corticosteroid treatment.

The primary endpoint was a comparison of results from the four-stair climb (4SC) assessment between baseline and 72 weeks. Secondary endpoints included function, strength, and muscle morphology.

Ultimately, patients treated with givinostat experienced an average 4SC time increase of just 1.25 seconds compared with 3.03 seconds in the placebo group. Secondary endpoints were more positive among patients treated with givinostat, yet not statistically significant.

The most common events in patients treated with givinostat included diarrhea, abdominal pain, thrombocytopenia, vomiting, hypertriglyceridemia, and fever. No treatment-related deaths were recorded.

Duvyzat will be available as an 8.86 mg/mL oral suspension. The drug should be prescribed by doctors experienced in treating DMD.

Detailed recommendations for using Duvyat will be described in the summary of product characteristics, which will be published in the European public assessment report in all official European Union languages.

Annie Lennon is a medical journalist. Her writing appears on Medscape Medical News, WebMD, and Medical News Today, among other outlets.