The European Medicines Agency has just pronounced favorably to the granting of an AMM with early access to Duvyzat, a specialty which made it possible to improve the motor function of children with Duchenne muscular dystrophy, after 18 months of treatment.
The European Medicines Agency (EMA) has recommended the granting of a parole (that is to say in early access) for Duvyzat (Givinostat) as a treatment for Duchenne muscular dystrophy in patients from six years capable of walking. Duvyzat is a oral suspension to take in combination with corticosteroid therapy.
This opinion is good news in the face of this pathology for which there is no curative treatment to date. “The treatment is based on corticosteroid therapy, the prevention of contractures and medical care of the cardiac and respiratory function”specifies the EMA.
The European Agency’s opinion is based on the data of a subgroup of 120 patients (79 treated by Givinostat and 41 by placebo) in a randomized study by placebo conducted with patients with muscular dystrophy of Duchenne ambulatory aged six years or more and receiving concomitant treatment with steroids. The study evaluated the modification of the time required to climb four steps (4SC), a tool widely used to assess the motor function, at 18 months. The results were statistically significant, the time required to climb four steps (4SC) on average only 1.25 seconds in patients treated with Givinostat, against 3.03 seconds in the placebo group. Other criteria have been evaluated such as muscle strength and morphology: their evolution was not statistically significant, but all the results were more positive in patients treated with Givinostat.
The most common side effects in Patients treated with Givinostat were diarrhea and abdominal pain, thrombocytopenia, vomiting, hypertriglyceridemia and fever.
Duchenne muscular dystrophy is a rare genetic disease that mainly affects boys. It is caused by a dystrophin deficit, a muscle protein. Over time, the muscles deteriorate, leading to difficulties in walking and a loss of muscle strength, then breathing difficulties that can lead to premature death. The life expectancy of people with this disease has increased over the years, some patients surviving beyond 30 years. Duvyzat’s active ingredient is Givinostat. It is an inhibitor of the Désacetylase histone (HDAC) which acts by targeting pathogenic processes to reduce inflammation and muscle cast iron. He was authorized in the United States in March 2024.
