A groundbreaking new drug for a severe muscle-wasting condition is set to be rolled out on the NHS in England, offering hope to hundreds of children. Givinostat, also known as Duvyzat and manufactured by ITF Pharma, has been approved for eligible patients with Duchenne muscular dystrophy (DMD) after its manufacturer struck a commercial deal with NHS England.
Hope for Families
The National Institute for Health and Care Excellence (Nice) confirmed the drug's availability, marking a significant step for families affected by this rare genetic disorder. Givinostat is expected to help patients maintain their mobility for longer, with trials suggesting it can increase the time before losing the ability to walk by around five years.
While campaigners welcomed the long-awaited approval, they highlighted the "agonising" two-year process during which many families were left without access as their child's condition deteriorated. Alex Clarke, whose 12-year-old son Ben can now access the drug, said: "It's been utterly devastating knowing there is a treatment out there that could slow down the progression of DMD, but not being able to access it."
About Duchenne Muscular Dystrophy
DMD is caused by a lack of the protein dystrophin, leading to muscle fibre breakdown. Primarily affecting boys, an estimated 2,005 individuals live with the condition in the UK. Symptoms typically emerge around age three, including difficulties with running, jumping, climbing stairs, or rising from the floor. Patients gradually lose muscle control, eventually losing the ability to walk or sit, and may need breathing assistance. Most reach adulthood but die before or during their 30s.
Eligibility and Access
Givinostat will be offered to DMD patients aged six or older who can still walk or stand. Nice estimates around 530 people in England will benefit. The drug has been available since 2024 via an early access programme, but families reported postcode-based access issues. Alex Clarke's local NHS trust did not participate, leaving his son without treatment until now.
Helen Knight, director of medicines evaluation at Nice, said: "We are extremely grateful to the patient representatives who gave powerful testimony... Givinostat is a promising treatment, so we are delighted the company and NHS England have been able to reach a deal."
Charity Response
Emily Reuben, co-founder and chief executive of Duchenne UK, hailed the approval as a "significant breakthrough" but stressed the need to end the "postcode lottery." Rosie Day, whose twin children have DMD, said: "For boys like our son Jasper, it gives us real hope for his future."
However, Alex Johnson, co-founder of Duchenne UK and chief executive of Joining Jack, expressed disappointment that access is restricted to those who can walk or stand, urging stakeholders to explore options for non-ambulant patients.
Cost and Funding
Givinostat costs about £250,000 per year per patient at list price, but a commercial access agreement with NHS England ensures a discounted rate. The drug will be funded immediately through the Innovative Medicines Fund, Nice confirmed.
