The world’s most expensive drug, which treats babies and young children with a rare and often fatal degenerative disorder, will be available this year for the first time on the NHS in England.
Zolgensma, which costs £1.79m per dose, halts the progression of spinal muscular atrophy (SMA), which involves loss of movement, muscle weakness and paralysis, and is the leading genetic cause of death in infants.
Up to 80 infants a year in England are expected to benefit from the drug, which is administered as a single dose and replaces the function of the faulty SMN1 gene. The active ingredient, onasemnogene abeparvovec, restores the gene and produces proteins essential for muscle movement and nerve function.
Babies born with the most severe form of the muscle-wasting disease – SMA type 1 – have a life expectancy of two years. Zolgensma does not cure the disease but helps halt its progression, meaning babies breathe without a ventilator, sit up, crawl and even walk, with benefits lasting more than five years after treatment.
For some babies diagnosed before developing symptoms it “might... see more