Around 530 people in England living with Duchenne muscular dystrophy (DMD) are set to benefit from a new treatment following the successful negotiation of a commercial deal to make givinostat (Duvyzat) available on the NHS.
The decision has been confirmed in final draft guidance, with the treatment made available immediately through the Innovative Medicines Fund, providing a new option for patients with this rare and progressive condition.
Duchenne muscular dystrophy is a rare genetic condition, primarily affecting boys, with symptoms usually appearing from around three years of age.
The disease is caused by the absence of dystrophin, a protein essential for maintaining muscle strength and function. Over time, muscle weakness worsens, leading to:
- Loss of ability to walk and stand
- Increasing dependency on carers
- Progressive damage to the heart and lungs
DMD is a severe, life‑limiting condition, with no cure currently available.
The new treatment will be available to patients aged six and over who are still able to walk or stand, with or without support, at the start of treatment.
This aligns with the patient group studied in clinical trials, rather than the full licensed population.
Givinostat is now the third treatment recommended for DMD, offering an additional option for managing the disease.
A key benefit of givinostat is its ability to delay the progression of Duchenne muscular dystrophy, regardless of the specific genetic type of the disease. Clinical evidence suggests that the treatment may help extend the length of time patients can walk by around five years on average compared with standard care.
Standard management typically includes corticosteroids and supportive therapies, but givinostat provides a new mechanism of action to improve outcomes.
Givinostat works by targeting biological processes that contribute to muscle damage and inflammation in DMD.
By modifying these pathways, the treatment helps to protect muscle function and slow deterioration, supporting patients to maintain independence for longer.
The medicine has been available in England since November 2024 through an early access programme, giving patients earlier access ahead of formal NHS approval.
While trial results are promising, NICE highlighted that there are limitations in the available evidence, including uncertainty about the long‑term impact of the treatment beyond delaying loss of mobility.
The evaluation committee recognised the challenges of collecting robust evidence in rare diseases such as DMD, particularly when measuring outcomes like quality of life for patients and their carers.
As a result, the committee accepted a higher level of uncertainty than usual, reflecting the seriousness of the condition and limited treatment options.
After reviewing the clinical and economic evidence, NICE concluded that givinostat represents a cost‑effective use of NHS resources, applying a maximum severity weighting of 1.7 in recognition of the condition’s impact.
This weighting reflects the significant burden of DMD on patients, families, and healthcare services, and allows for greater flexibility in decision‑making for treatments targeting severe conditions.
By slowing disease progression, givinostat is expected to help:
- Extend periods of mobility and independence
- Reduce complications associated with muscle deterioration
- Improve overall quality of life for patients
The treatment may also benefit families and carers by delaying the point at which full-time support is required.
Director of Medicines Evaluation at NICE, Helen Knight, commented:
“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.
"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."
With funding secured through the Innovative Medicines Fund, eligible patients in England will be able to access givinostat straight away, avoiding delays often associated with new treatments.
The approval of givinostat marks a significant step forward in the treatment of Duchenne muscular dystrophy, offering new hope to patients and their families.
While further evidence is needed to fully understand its long‑term benefits, the drug provides a valuable new option in a condition where treatment choices have historically been limited.
By combining early access with ongoing data collection, the NHS aims to continue improving outcomes for patients living with this challenging and life‑limiting condition.
Image credit: iStock
