The Medicines and Healthcare products Regulatory Agency (MHRA) has approved Italfarmaco’s Duvyzat (givinostat) to treat Duchenne muscular dystrophy (DMD) in patients aged six years and older.
The regulator has specifically granted full approval of the drug for patients who are beginning treatment when ambulatory, and has issued a conditional approval for those who start treatment when no longer ambulatory.
Estimated to affect approximately 2,500 people in the UK at any one time, DMD is a rare neuromuscular disease caused by a change or mutation in the gene that encodes instructions for dystrophin, which is required to strengthen and protect muscles.
Symptoms of the disorder are usually first seen in boys aged two five years and worsen over time, affecting patients’ ability to walk as well as their heart and respiratory muscles.
Duvyzat is an orally-administered histone deacetylase inhibitor that works by targeting pathogenic processes to reduce inflammation and loss of muscle.
The MHRA’s decision references the US Food and Drug Administration’s approval of the drug in March and was granted through its International Recognition Procedure, which allows the UK regulator to take into account the decision-making of trusted regulatory partners.
Supporting both applications were positive results from the late-stage EPIDYS trial of 179 ambulant boys, who were randomised to receive either Duvyzat twice daily or placebo, in addition to glucocorticosteroid treatment.
The study met its primary endpoint, with Duvyzat-treated patients demonstrating a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment.
Treatment with Duvyzat was also associated with favourable results on key secondary endpoints, including North Star Ambulatory Assessment, a scale commonly used to rate the motor function in boys with DMD who are capable of walking.
Paolo Bettica, chief medical officer, Italfarmaco Group, said: “This approval is based on our robust, clinically meaningful, successful development programme in one of the largest DMD trials to-date… Our focus now is to make Duvyzat available as a treatment for DMD management in the UK as quickly as possible.”
The launch of Duvyzat in the UK will be led by Italfarmaco’s UK subsidiary, ITF Pharma UK.