MHRA Approves Duvyzat for Duchenne Muscular Dystrophy Treatment

The Medicines and Healthcare products Regulatory Agency (MHRA) has given the green light to Italfarmaco's Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy (DMD) in patients who are six years of age and older. This approval marks a significant step forward in managing this progressive neuromuscular disorder. The MHRA's decision includes full approval for patients who commence treatment while still able to walk, and a conditional approval for those who begin treatment after they have lost the ability to walk.

Duchenne muscular dystrophy is a serious condition that affects approximately 2,500 individuals across the United Kingdom at any given time. It is caused by mutations in the gene responsible for producing dystrophin, a protein essential for muscle strength and protection. Symptoms of DMD typically manifest in boys between the ages of two and five and progress over time, eventually impacting their mobility as well as cardiac and respiratory muscles.

Duvyzat is an oral medication that acts as a histone deacetylase inhibitor. Its function is to target and reduce the pathogenic processes that lead to inflammation and muscle deterioration, thereby addressing some of the underlying causes of DMD. The MHRA's decision was influenced by the prior approval of the drug by the US Food and Drug Administration (FDA) in March. This decision was facilitated through the MHRA's International Recognition Procedure, which allows it to consider the evaluations made by other respected regulatory authorities.

Supporting the approval were promising outcomes from the late-stage EPIDYS clinical trial, which involved 179 ambulant boys who were randomly assigned to receive either Duvyzat twice daily or a placebo, in conjunction with glucocorticosteroid therapy. The trial achieved its primary objective, demonstrating that patients treated with Duvyzat showed a significant and clinically relevant improvement in their time to complete the four-stair climb test.

Furthermore, treatment with Duvyzat yielded positive results on crucial secondary measures, including the North Star Ambulatory Assessment. This assessment is a widely-used tool for evaluating motor function in boys with DMD who retain the ability to walk.

Paolo Bettica, the chief medical officer of the Italfarmaco Group, expressed enthusiasm about the approval, noting the robust and clinically significant data from their comprehensive development program, which is considered one of the largest DMD trials conducted to date. He emphasized the group's commitment to making Duvyzat available as swiftly as possible for DMD management in the UK.

The introduction of Duvyzat into the UK market will be spearheaded by ITF Pharma UK, a subsidiary of Italfarmaco. This development holds promise for improving the quality of life for those affected by Duchenne muscular dystrophy and represents a noteworthy advancement in treatment options for this challenging disease.