In June, five-month-old Arthur Morgan became the first person in the UK to be treated with one-off gene therapy Zolgensma, manufactured by Novartis. A single dose of the potentially life-saving therapy for spinal muscular atrophy (SMA) is an astonishing £1.795m, making Zolgensma the most expensive drug in the world.
Untreated SMA, which causes muscle weakness and paralysis, is the leading genetic cause of death in children. Zolgensma treats the root cause of the disease; clinical evidence suggests it can significantly improve motor function in young patients.
The drug’s approval was a milestone for families with SMA, but it does raise the question: can pharmaceutical companies justify such hefty prices for their medicines?