A groundbreaking drug for Duchenne muscular dystrophy (DMD) has been approved for use on the NHS in England, offering new hope to patients with this severe muscle-wasting condition. The medication, known as Givinostat and marketed as Duvyzat, received the green light following a commercial agreement between its manufacturer and the National Institute for Health and Care Excellence (Nice).
Eligibility and Impact
The treatment will be available to DMD patients aged six and older who retain the ability to walk or stand. Clinical trials suggest that Duvyzat could extend mobility by approximately five years for around 530 individuals in England. Duchenne muscular dystrophy is a rare genetic disorder that primarily affects boys, caused by a lack of the protein dystrophin, leading to progressive muscle degeneration, loss of mobility, and often premature death.
Reactions from Families and Campaigners
Families affected by the condition have welcomed the approval but expressed frustration over the delays. Alex Clarke, whose son Ben will now receive the drug, described the two-year wait as 'agonising' and criticised the previous 'postcode lottery' during an early access programme. Alex Johnson, co-founder of Duchenne UK, voiced disappointment that access is restricted to those who can still walk, urging stakeholders to explore options for patients who have already lost mobility. Despite these concerns, the drug will be funded immediately through the Innovative Medicines Fund.
