A new drug that can slow the progression of Duchenne muscular dystrophy (DMD) is now available on the NHS for children and young people, offering hope to hundreds of families across England. The treatment, Givinostat, has been approved for use after the drug’s manufacturer, ITF Pharma, reached a commercial agreement with NHS England, the National Institute for Health and Care Excellence (Nice) has confirmed.
The decision means eligible patients aged six and over who can still walk or stand, with or without support, will be able to receive the drug, which is expected to help them maintain their mobility for longer. Nice estimates that around 530 people in England will benefit from the rollout. The drug will be funded immediately through the Innovative Medicines Fund, with a list price of approximately £250,000 per patient per year, though the commercial agreement secures it at a discounted rate.
How Givinostat slows the progression of Duchenne muscular dystrophy
Duchenne muscular dystrophy is a rare, genetic, progressive and fatal condition caused by a lack of the protein dystrophin, which is essential for muscle fibre stability. It primarily affects boys, with an estimated 2,005 individuals living with the condition in the UK and around 100 boys born with it each year. Symptoms typically begin around the age of three, manifesting as difficulties with running, jumping, climbing stairs, or rising from the floor. Over time, patients gradually lose control of their muscles, losing the ability to walk or sit, and may eventually need mechanical assistance to breathe. Most people with DMD reach adulthood but are likely to die before or during their 30s.
Givinostat, marketed as Duvyzat, is a histone deacetylase (HDAC) inhibitor. It works by targeting the pathogenic processes that drive inflammation and muscle loss, and by regulating the overactive HDAC activity in dystrophic muscle tissue — a direct consequence of dystrophin deficiency. This mechanism helps counteract the disease pathology, slow muscle degradation, and promote muscle regeneration. It is the first nonsteroidal drug approved for all genetic variants of DMD.
Clinical trial evidence suggests that Givinostat can extend the period before patients lose the ability to walk by approximately five years. Long-term data from the EPIDYS trials indicate that when used alongside corticosteroids, the drug significantly delayed disease progression in ambulant patients aged six and older. Specifically, it delayed the loss of the ability to rise from the floor by a median of two years, the ability to perform a four-stair climb by 3.3 years, and the maintenance of ambulation by 2.9 years. The drug is administered orally, typically twice daily with food.
As with any medicine, Givinostat carries potential side effects. According to trial data, common adverse reactions include gastrointestinal issues such as diarrhoea, abdominal pain, nausea, and vomiting. Other reported effects include thrombocytopenia (low platelet count), hypertriglyceridemia (elevated blood fat levels), fever, muscle or joint pain, fatigue, and decreased appetite. Serious side effects can include severe headache, confusion, vision problems, and heart rhythm abnormalities such as QT prolongation. Regular monitoring of platelet counts and triglyceride levels is recommended during treatment.
Givinostat received conditional marketing authorisation from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) on 20 December 2024, and had already been approved by the US Food and Drug Administration in March 2024. Nice’s final draft guidance approving its use on the NHS in England was published on 7 May 2026, following approval for NHS use in Scotland in December 2025. The drug had been available through an early access programme (EAP) since November 2024, but families reported significant difficulties accessing the treatment based on where they lived. Some NHS trusts delayed or refused access, citing the cost of monitoring — estimated at around £1,900 per patient per year — as a barrier, since the EAP covered the drug but not associated delivery and staffing costs.
‘A long time coming’: patient and charity reactions
Alex Clarke, whose local NHS trust did not take part in the early access programme, said he was “delighted” that his 12-year-old son Ben can now access Givinostat. “Today’s decision has been a long time coming for our family,” he said. “It’s been utterly devastating knowing that there is a treatment out there that could slow down the progression of DMD and give Ben more time to do the things he loves, but not being able to access it. We are of course delighted that Ben will now be able to access this new medicine through the NHS, but it should never have taken this long and it’s vital that he can now do so without any further delay.”
Rosie Day, from Horsham in West Sussex, has twin children with DMD: Jasper and Arabella, the latter a carrier of the genetic disorder while Jasper is the most affected. “We have waited a long time for this decision and it is an important day for the whole Duchenne community,” she said. “For boys like our son, Jasper, it gives us real hope for his future. It will be key that all boys can access it as soon as possible.”
Helen Knight, director of medicines evaluation at Nice, said: “We are extremely grateful to the patient representatives who gave powerful testimony to the committee. They described the serious impact of Duchenne muscular dystrophy on patients and their families and the importance of givinostat as a treatment option, giving hope where there is currently none. Givinostat is a promising treatment, so we are delighted the company and NHS England have been able to reach a deal that recognises its potential but also the uncertainties that remain around the extent of the benefit it provides.”
The charity Duchenne UK welcomed the approval, with co-founder and chief executive Emily Reuben hailing it as a “significant breakthrough” for people with the condition. Ms Reuben, whose eldest son has DMD, said: “We hope today’s decision will make this treatment available swiftly to all eligible patients in England and end the postcode lottery of access that families have faced across the UK.”
Duchenne UK has been supporting families through its Time is Muscle campaign, which started in November 2024, and met with Health Secretary Wes Streeting in January 2025 to speed up access. Alex Johnson, co-founder of Duchenne UK and chief executive of Joining Jack — a charity set up after her son Jack’s diagnosis in 2011 — said: “While we celebrate givinostat’s approval today, it has been a long, agonising process to get us to this point. It has taken Nice almost two years to make a decision, during which time many families were left without access to this medicine, while their child’s condition progressed and they lost mobility and function, which once lost, can never be regained in DMD.”
Ms Johnson also expressed disappointment that access to Givinostat is restricted to certain patients. “We are disappointed that this approval is restricted to people with Duchenne who can walk or stand, meaning that anyone who is unable to walk or stand will not be given access under this guidance. We are urging the relevant stakeholders to look at all available options to make givinostat available to these patients, who desperately need new treatment options.”