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Approval of Duvyzat (givinostat) for Duchenne muscular dystrophy in the USA

22 March 2024

On Thursday, 21 March 2024, the oral drug for the treatment of Duchenne muscular dystrophy (DMD), Duvyzat (also known as givinostat), was approved by the FDA (U.S. Food and Drug Administration) for use in boys aged 6 and older in the United States. Duvyzat is the first non-steroidal drug approved to treat people with all genetic variations of DMD.

The news follows on from Italfarmaco Group announcing results from the EPIDYS clinical trial earlier this week. EPIDYS was a Phase 3, randomised, double-blind, placebo-controlled clinical trial for givinostat. Phase 3 clinical trials assess how efficient and safe a tested drug is among the participants.

The results published in The Lancet Neurology journal show that the study met its primary objective (also known as endpoint). The primary endpoint was to demonstrate a better outcome in the four-stair climb assessment from baseline (starting point, before the treatment was given) to 72 weeks later, against those who received the placebo.

The trial enrolled 179 ambulant boys with DMD (who can walk unassisted) aged 6 and older. Italfarmaco reported a slower decline in performing functional tasks in boys who were given givinostat compared to those who received a placebo. This showed that givinostat can delay disease progression when combined with corticosteroid treatment.

FDA approvals do not affect the UK. The health authority in the UK responsible for issuing Marketing Authorisations for medicines is the Medicines & Healthcare Products Regulatory Agency (MHRA). ITF Pharma Ltd (Italfarmaco Group in the UK) is engaging with the MHRA on securing a licence for givinostat. In addition to gaining a licence, further assessments of clinical and cost-effectiveness by the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) must also be undertaken before a medicine is available to patients on the NHS.

Duchenne muscular dystrophy is a rare, muscle-wasting condition that mainly affects boys and men. People with DMD lack a protein called dystrophin, which is required to maintain the strength of muscles. Givinostat is a drug from a family of molecules called HDAC (histone deacetylase) inhibitors that aims to restore muscle regeneration, increase muscle mass, and reduce inflammation and scar tissue accumulation in boys with DMD. 

This is an exciting step forward for the community and we will keep you updated on developments that impact people in the UK.

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