HQ Team
April 27, 2025: Italfarmaco SpA., a privately held Italian pharmaceutical and chemical company, received a recommendation for the conditional marketing in the European Union of an oral drug for Duchenne muscular dystrophy (DMD) in patients six years or older, who can walk.
The drug Duvyzat is an oral suspension to be taken at the same time as corticosteroid treatment.
A conditional marketing authorisation is one of the EU’s regulatory mechanisms to facilitate early access to medicines that fulfil an unmet medical need.
This type of approval allows the agency to recommend a medicine for marketing authorisation with less complete data than normally expected, if the benefit of a medicine’s immediate availability to patients outweighs the risk inherent in the fact that not all the data are yet available.
Muscles weaken, lose function
Duchenne muscular dystrophy is a rare genetic disease caused by a lack of dystrophin, a protein that helps strengthen muscle fibres and protect them from injury as muscles contract.
This causes the muscles to progressively weaken and lose function, and is ultimately lethal. This process is worsened by increased levels of histone deacetylases (HDAC), a class of proteins.
The recommendation was based on opinion from the Committee for Medicinal Products for Human Use (CHMP), a committee within the European Medicines Agency (EMA) responsible for evaluating and providing opinions on human medicines intended for use in the European Union, according to an agency statement.
The opinion was based on data from a subgroup of 120 patients — 79 treated with givinostat and 41 with a dummy drug — in a study in ambulant Duchenne muscular dystrophy patients aged six years or older on concomitant steroid treatment.
Primary, secondary goals
The primary endpoint was the change in time to complete a four-stair climb, a tool widely used to assess motor function, at 18 months.
The four-stair climb time increased by an average of only 1.25 seconds in patients treated with givinostat compared to 3.03 seconds in the placebo group, according to a company statement.
The difference in the key secondary endpoints assessing function, strength and muscle morphology was “not statistically significant,” but all outcomes were more positive in patients treated with givinostat, according to the EMA statement
There is no cure for Duchenne muscular dystrophy, and treatment is centred on corticosteroid therapy, prevention of contractures, and medical care of the heart and respiratory function.
The active substance of Duvyzat is givinostat, an HDAC inhibitor that modulates uncontrolled HDAC activity in dystrophic muscles. Inhibiting HDAC activity may help reduce inflammation and tissue scarring, and thickening.
Conduct another study
The safety profile of Duvyzat is based on data from 179 patients. The most common events in patients treated with givinostat were diarrhoea, abdominal pain, thrombocytopenia (low levels of blood platelets), vomiting, hypertriglyceridaemia (high blood levels of triglycerides, a type of fat) and fever.
To confirm the efficacy of Duvyzat, the company has been requested to conduct a study in ambulant DMD patients and a post-authorisation efficacy and safety study based on data from a patient registry of children with DMD six years and older treated with givinostat.
Duvyzat was designated as an orphan medicinal product on July 4, 2012, for the treatment of Duchenne muscular dystrophy.
Following this positive CHMP opinion, the Committee for Orphan Medicinal Products (COMP) will assess whether the orphan designations should be maintained, according to the statement.
