Hundreds of children across England are set to benefit from a new drug which has been approved for rollout on the NHS to treat a severe muscle-wasting condition.
Givinostat is expected to enable eligible patients with Duchenne muscular dystrophy (DMD) to maintain their mobility for longer.
The National Institute for Health and Care Excellence (Nice) confirmed the drug’s availability after its manufacturer reached a commercial agreement with NHS England.
This decision marks a significant step for families affected by the rare genetic disorder.
While campaigners welcomed the long-awaited approval, they highlighted the “agonising” two-year process, during which many families were left without access to the drug as their child’s condition continued to deteriorate.
Duchenne muscular dystrophy is caused by a lack of the protein dystrophin, leading to the breakdown of muscle fibres.
Primarily affecting boys, an estimated 2,005 individuals live with the condition in the UK.
Symptoms typically emerge around the age of three, manifesting as difficulties with running, jumping, climbing stairs, or rising from the floor.
Patients gradually lose control of muscles, losing the ability to walk or sit, and can eventually need help from machines to breathe.
Most people with the condition reach adulthood, but are likely to die before or during their 30s.
Givinostat, known as Duvyzat and made by ITF Pharma, will be offered to DMD patients aged six or over who can still walk or stand.
Trials suggest that the drug can increase the length of time people have before losing the ability to walk by around five years.
Nice estimates that around 530 people in England will benefit.
The drug has been available on the NHS since 2024 as part of an early access programme, but families have reported struggling to access the treatment based on where they live.
Alex Clarke, whose local NHS trust did not take part in the early access programme, said he was “delighted” 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.”
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.”
Rosie Day has twin children with DMD, Jasper and Arabella.
Arabella is a carrier of the genetic disorder and Jasper is the most affected.
Mrs Day, from Horsham in West Sussex, who has two older daughters, told the Press Association: “We have waited a long time for this decision and it is an important day for the whole Duchenne community.
“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.”
Givinostat costs about £250,000 per year per patient at its list price, although its manufacturer has struck a commercial access agreement with the NHS which makes it available with a discount.
Duchenne UK has been supporting families through its Time is Muscle campaign, which started in November 2024, and met with Health Secretary Wes Streeting last January in a bid to speed up access.
Alex Johnson is the co-founder of Duchenne UK and chief executive of Joining Jack, a charity which was set up following her son Jack’s diagnosis of DMD in 2011.
She 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 said she is “disappointed” 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,” she added.
“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.”
Givinostat will be funded immediately through the Innovative Medicines Fund, Nice said.