NICE has approved givinostat for routine NHS use in England after a commercial deal was reached, giving around 530 people access to a new treatment for Duchenne muscular dystrophy. The decision, announced on 8 May 2026, marks the third treatment NICE has recommended for the condition and means the medicine will be funded immediately through the Innovative Medicines Fund.
A new option for a severe and progressive condition
Givinostat, also known as Duvyzat and made by ITF Pharma, can be used in people aged 6 and over who are able to walk or stand, with or without support, at the start of treatment. NICE said this reflects the populations studied in clinical trials rather than the full licensed population. The medicine is intended to slow disease progression in Duchenne muscular dystrophy, a rare condition that mainly affects boys.
Duchenne muscular dystrophy usually begins to show symptoms at around age 3 and is caused by the lack of dystrophin, a protein that is important for muscle cells. NICE described it as a severe, rare, progressive and fatal condition. Over time, people with the disease gradually lose control of their muscles, lose the ability to walk and then sit, become quadriplegic and fully dependent on carers, before the illness affects the heart and lungs and eventually requires help from machines to breathe.
What the evidence showed
According to NICE, evidence from clinical trials suggests givinostat may increase the length of time people with Duchenne muscular dystrophy can walk by around five years on average compared with established clinical management, which includes corticosteroids and supportive care. However, NICE said limitations in the evidence mean it is not yet known how much benefit the treatment provides beyond that estimate.
Helen Knight, director of medicines evaluation at NICE, said the deal recognises the medicine’s potential while also acknowledging the remaining uncertainty about the size of the benefit. She said the recommendation means the treatment can now be made available for around 530 people across England living with the condition.
Families and clinicians welcomed the decision
NICE said patient representatives gave powerful testimony to the committee about the impact of Duchenne muscular dystrophy on patients and families and the importance of having another treatment option. Health Innovation and Safety Minister Dr Zubir Ahmed said the approval shows the NHS can secure available treatments and make them accessible quickly and fairly.
NICE added that the committee accepted a greater level of uncertainty than usual because of the difficulties in collecting evidence on both effectiveness and quality of life. Taking those factors into account, and applying the maximum 1.7 severity weighting, the committee concluded that givinostat would be a cost-effective use of NHS resources.
The drug has been available since November 2024 under an early access programme, but the new guidance now opens the way for broader NHS use in England. For families affected by Duchenne muscular dystrophy, the decision offers a new treatment option in a disease where progress has long been limited and the outlook remains serious.
Source: NICE news release
