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

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.