The European Medicines Agency (EMA) has suggested that Duvyzat (givinostat) be given a conditional marketing authorization in the EU to treat Duchenne muscular dystrophy (DMD) in individuals who can walk starting at age six. Duvyzat is a suspension that is given orally in conjunction with corticosteroid therapy.
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Is Duvyzat a Treatment for DMD?
There is no cure for DMD and treatment is centred on corticosteroid therapy, prevention of contractures, and medical care of the heart and respiratory function. (What is Duvyzat (givinostat)?)
Is Duvyzat Safe?
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.
Duvyzat Approved in Europe
Duvyzat is recommended for a conditional marketing authorisation, 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.
In order to confirm the efficacy of Duvyzat, the company has been requested to conduct a randomised, placebo-controlled 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.
The opinion adopted by the CHMP is an intermediate step on Duvyzat’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role/use of this medicine in the context of the national health system of that country.
