NICE recommends Duvyzat for Duchenne muscular dystrophy

NICE has backed routine NHS use of Duvyzat (givinostat) for Duchenne muscular dystrophy in England, opening up access to around 530 eligible patients after a commercial agreement with the manufacturer. Duvyzat developed by ITF Pharma, is recommended for people aged six and over who can still walk or stand, with or without support, at the start of therapy, mirroring the population studied in clinical trials rather than the full licensed population.

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Under the final draft guidance, givinostat will be funded immediately through the Innovative Medicines Fund and becomes the third DMD treatment endorsed by NICE, offering an option that can slow disease progression regardless of the underlying genetic subtype.

The drug has already been available via an early access programme since late 2024, but this decision is expected to standardise availability across England. DMD, which mainly affects boys and typically presents around age three, is a severe, progressive and ultimately fatal neuromuscular condition caused by an absence of dystrophin, leading to loss of mobility, dependence on carers, and eventual respiratory and cardiac failure.

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Trial evidence suggests givinostat could extend the period patients are able to walk by roughly five years on average when added to standard care with corticosteroids and supportive management, although uncertainties remain around the full magnitude and duration of benefit.

NICE’s committee acknowledged the challenges of generating robust data in a rare, rapidly progressive disease and accepted a higher-than-usual level of uncertainty; after applying the maximum severity weighting of 1.7, it concluded that givinostat represents a cost-effective use of NHS resources.