WIREDGOV

Around 530 people in England will benefit from access to the Duchenne muscular dystrophy treatment givinostat, following the successful negotiation of a commercial deal to make the medicine available on the NHS.

Today’s final draft guidance says givinostat (also called Duvyzat and made by ITF Pharma) can be used to treat Duchenne muscular dystrophy (DMD) in people aged 6 and over who are able to walk or stand, with or without support, at the start of treatment. This is in line with the populations studied in clinical trials rather than the full licensed population.

Givinostat will be funded immediately through the Innovative Medicines Fund and becomes the third treatment for the condition we’ve recommended. Significantly, it provides an option for people with DMD that can delay the progression of the disease irrespective of the type of DMD they have.

Givinostat has been available since November 2024 under an early access programme.

Affecting mostly boys, with symptoms that usually appear at around age 3, DMD is one of a group of muscular dystrophies caused by the lack of a protein called dystrophin which is important for muscle cells. It is a severe, rare, progressive and fatal condition.

People with DMD gradually lose control of muscles, meaning they lose the ability to walk, and then sit, becoming quadriplegic and completely dependent on carers. The disease progresses until it affects the heart and lungs and eventually, they need help from machines to breathe.

Evidence from clinical trials suggests that givinostat may increase the length of time people with DMD can walk by around 5 years on average compared with established clinical management, which includes treatment with corticosteroids and supportive care. However, because of limitations in the evidence, it is not yet known how much benefit givinostat provides beyond that.

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. In doing so it means NICE can recommend the treatment for around 530 people across England with this devastating condition.

Helen Knight, director of medicines evaluation at NICE

Helen continues, "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."

Duchenne muscular dystrophy is a cruel condition that robs children of their mobility and independence and causes immense heartache for families. Thanks to action by this government, this treatment has been approved for the first time - giving patients the power to slow the progression of this devastating disease.

Health Innovation and Safety Minister, Dr Zubir Ahmed

Dr Ahmed added, "This is exactly what our NHS is here to do: secure the best available treatments for patients and ensure they are available quickly and fairly. I want to thank everyone involved in reaching this commercial deal, and most of all the families who shared their experiences to help make this possible."

The committee recognised the difficulties in collecting evidence on both givinostat’s effectiveness and its impact on the quality of life for people with DMD and their carers meant allowing a greater level of uncertainty than usual was appropriate. Taking all these 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.