NHS England to Roll Out Givinostat for Eligible Duchenne Muscular Dystrophy Patients

Hundreds of children in England will gain access to a drug designed to slow the progression of Duchenne muscular dystrophy after its manufacturer struck a commercial deal with NHS England. The National Institute for Health and Care Excellence confirmed that givinostat will be made available to eligible patients.

The drug, known as Duvyzat and produced by ITF Pharma, is expected to help maintain mobility for longer in those who can still walk or stand. Duchenne muscular dystrophy is caused by a lack of the protein dystrophin, which leads to the breakdown of muscle fibres.

The condition primarily affects boys, with an estimated 2,005 individuals living with it in the UK. Symptoms usually appear around age three, including difficulties with running, jumping, climbing stairs or rising from the floor. Patients progressively lose muscle control, eventually losing the ability to walk or sit unaided and may require mechanical ventilation.

Most reach adulthood but are likely to die before or during their 30s.

Givinostat will be offered to patients aged six and over who can still walk or stand. Clinical trials indicate the drug can extend the time before loss of walking ability by around five years. The National Institute for Health and Care Excellence estimates that around 530 people in England will benefit.

The treatment has been available on the NHS since 2024 through an early access programme. Families have reported inconsistent access depending on where they live. A parent whose local NHS trust did not participate in the early access programme said the decision had been a long time coming.

The parent described the period without access as devastating given the progressive nature of the condition.

Givinostat costs about £250,000 per patient per year at list price. The manufacturer has agreed a discounted commercial access arrangement with NHS England. Funding will begin immediately through the Innovative Medicines Fund. The National Institute for Health and Care Excellence director of medicines evaluation stated that patient representatives provided testimony about the impact of the condition and the importance of the treatment option.

The director noted that the agreement recognises the drug's potential while acknowledging remaining uncertainties about the extent of its benefit. A charity supporting families with the condition described the approval as a significant breakthrough.

The group expressed hope that the treatment would become available swiftly to all eligible patients in England and address regional differences in access that families have faced. Another family affected by the condition, with one child showing severe symptoms, said the decision provided real hope.

The family emphasised the need for rapid access for all eligible boys. A representative from a Duchenne muscular dystrophy charity noted that the approval process had taken nearly two years. During that period some families lacked access while their children's condition progressed and they lost mobility and function that cannot be regained.

The representative expressed disappointment that access remains restricted to those who can still walk or stand and urged stakeholders to explore options for patients who no longer meet that criterion.