Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy. DMD is characterized by progressive muscle weakness leading to loss of ambulation, respiratory and cardiac impairment.
To show the possible effects of Givinostat, a pan HDAC inhibitor, on disease milestones, in DMD boys treated for more than 6 years.
We examined the long-term effects of Givinostat on milestone related to DMD disease progression such as, Loss of Ambulation and on Respiratory Function. Boys were assessed on motor and pulmonary function tests every 3 months. The safety and tolerability of long-term administration of Givinostat was evaluated as well.
Eighteen DMD patients enrolled and treated with Givinostat and corticosteroids for more than 6 years. Overall, Givinostat demonstrated a good tolerability profile over more than 6 years of treatment. The mean age at which they reached the Loss of Ambulation was 15.3 year.
After 64 months of treatment, a 2.0% yearly decline in FVC% Predicted was seen, while PEF% predicted was maintained.
The boys are still on treatment in a long-term safety trial.
Compared to the published natural history data, Givinostat administration appears to be associated with a slowdown of the disease progression. The EPIDYS trial, a phase III, randomized, double blind, placebo controlled trial is currently ongoing in Europe and North America in ambulant children older than or equal to 6 years of age to further explore Givinostat’s efficacy in DMD.