- Givinostat, a new drug for Duchenne muscular dystrophy (DMD), has been approved for rollout on NHS England following a commercial agreement between its manufacturer and the National Institute for Health and Care Excellence (Nice).
- The treatment, known as Duvyzat, will be available to eligible DMD patients aged six or over who retain the ability to walk or stand, with trials indicating it could extend mobility by approximately five years for around 530 individuals in England.
- Duchenne muscular dystrophy is a severe, rare genetic disorder primarily affecting boys, characterised by muscle breakdown due to a lack of dystrophin, leading to progressive loss of mobility and often premature death.
- While campaigners and families, such as Alex Clarke whose son Ben will now receive the drug, welcomed the approval, they criticised the 'agonising' two-year delay in access and the previous 'postcode lottery' for treatment during an early access programme.
- Duchenne UK co-founder Alex Johnson expressed disappointment that the drug's access is restricted to patients who can still walk, urging stakeholders to explore options for those who have already lost mobility, despite the drug being funded immediately via the Innovative Medicines Fund.
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