Access to treatments

Duchenne UK launches campaign to urge the MHRA to recognize FDA’s decision on Duvyzat

This morning, the United States Food and Drug Administration (FDA) granted approval to Duchenne muscular dystrophy (DMD) treatment Duvyzat (the brand name for givinostat). This gives the UK regulators, the Medicines and Healthcare products Regulatory Agency (MHRA), the opportunity to swiftly recognize and approve Duvyzat for patients age 6 years and older in the UK.

Approval in US

It has been approved for people with DMD who are six years of age and older, and for all genetic variants of DMD.

It will be available and marketed in the United States by Italfarmaco Therapeutics.

Givinostat

Givinostat is an ‘HDAC inhibitor’. It blocks enzymes called histone deacetylases (HDACs), which are involved in turning genes ‘on’ and ‘off’ within cells. It works by targeting pathogenic processes to reduce inflammation and muscle loss.

Results from the Italfarmaco Pivotal Phase 3 EPIDYS Study of givinostat in DMD were published in The Lancet Neurology this week. The EPIDYS clinical trial met its primary endpoint: change in four-stair climb assessment from baseline to 72 weeks, which demonstrated the potential of givinostat to delay disease progression when added to corticosteroid treatment. Givinostat-treated boys also showed favorable outcomes on key secondary endpoints assessed in the study.

What it means for people living with DMD in the UK

This is good news for the DMD community in the UK. Italfarmaco can use this approval to seek a quicker approval by the MHRA in the UK.

In the UK, currently, companies can take advantage of the UK’s International Recognition Procedure, which has been put in place to support the MHRA speeding up its review of treatments approved by trusted partners such as the FDA.

While it won’t lead to automatic approval of treatments approved by the MHRA, it can support reducing timelines for MHRA approval, as the agency can rely on the FDA feedback and opinion to inform their decision.

What we are doing

Duchenne UK is launching a campaign to urge the MHRA to recognise the FDA’s decision as soon as possible. Please sign up BELOW to be part of the campaign.