Hundreds of children across England will now receive a new drug for a severe muscle-wasting condition, after the National Institute for Health and Care Excellence (Nice) confirmed its availability on the NHS. Givinostat, manufactured by ITF Pharma and sold under the brand name Duvyzat, has been approved for eligible patients with Duchenne muscular dystrophy (DMD) following a commercial agreement between the company and NHS England.
The decision ends a lengthy appraisal process that had left many families without access to the treatment for almost two years. Nice estimates that around 530 people in England will benefit from the drug, which will be funded immediately through the Innovative Medicines Fund.
How givinostat helps patients maintain mobility
Givinostat is an HDAC inhibitor that works by targeting the pathogenic processes behind Duchenne muscular dystrophy, reducing inflammation and slowing the loss of muscle fibres. The condition is caused by a lack of the protein dystrophin, which leads to the progressive breakdown of muscle tissue. By addressing these underlying mechanisms, the drug aims to help patients hold on to their mobility for longer.
Clinical trial data indicates that patients taking givinostat maintained more muscle strength and motor function compared with those on a placebo. The drug also reduced the amount of new fat accumulating in muscle, a key measure of muscle deterioration. Trials suggest that givinostat can increase the length of time before a patient loses the ability to walk by approximately five years.
Under Nice’s guidance, the drug will be offered to DMD patients aged six or over who can still walk or stand, with or without support. This aligns with the populations studied in the clinical trials. At its list price, givinostat costs around £250,000 per year per patient, but the manufacturer has struck a commercial access agreement with NHS England that makes it available at a discounted price.
Givinostat has been available on the NHS since 2024 as part of an early access programme, but families reported significant difficulties accessing the treatment depending on where they lived. Some NHS trusts delayed or refused to take part, citing the cost of monitoring the drug — estimated at roughly £1,900 per patient annually — leading to what campaigners described as a “postcode lottery”.
Duchenne muscular dystrophy primarily affects boys, with around 2,005 individuals living with the condition in the UK. Approximately 100 boys are born with DMD in the UK each year, and around 20,000 children are born with the condition globally annually. Symptoms typically emerge around the age of three, including difficulties with running, jumping, climbing stairs, or rising from the floor. Patients gradually lose control of their muscles, eventually losing the ability to walk or sit, and can require mechanical assistance to breathe. Most people with the condition reach adulthood, but are likely to die before or during their 30s.
Families and charities react to the long-awaited approval
Charities and patient families have welcomed the decision but expressed frustration at the time it took to reach a conclusion. Alex Clarke, whose 12-year-old son Ben was unable to access givinostat through the early access programme because his local NHS trust did not take part, said he was “delighted” but that the wait had been “utterly devastating”.
“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,” he said. “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, who is the most affected, and Arabella, who is a carrier of the genetic disorder. “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, thanked 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,” she said. “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, which launched its “Time is Muscle” campaign in November 2024 and met with Health Secretary Wes Streeting last January in a bid to speed up access, hailed the approval as a “significant breakthrough”. Emily Reuben, co-founder and chief executive, 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.”
However, concerns remain about the scope of the approval. Alex Johnson, co-founder of Duchenne UK and chief executive of the charity Joining Jack, noted that the guidance restricts access to patients who can walk or stand, excluding those who have already lost mobility. “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,” she said. She urged all relevant stakeholders to look at available options to make givinostat available to non-ambulatory patients, “who desperately need new treatment options”.
Johnson also criticised the length of the Nice appraisal process. “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,” she said.
Elsewhere in the UK, the Scottish Medicines Consortium had already approved givinostat for NHS use in Scotland in December 2025. The European Medicines Agency recommended a conditional marketing authorisation for Duvyzat in the EU in April 2025, and the US Food and Drug Administration approved the drug in March 2024. Despite these steps, families in England have waited until now for routine access through the NHS.
