NHS treats first patient with the ‘world’s most expensive drug’

A five-month old baby has become the first patient to receive a potentially life-saving drug on the NHS that can prevent paralysis and prolong the lives of children with Spinal Muscular Atrophy.

Arthur Morgan, who was diagnosed with SMA earlier this month, received the one-off gene therapy at Evelina London Children’s Hospital on 25 May.

Untreated SMA is the leading genetic cause of death for children, and until two years ago there were no treatment options available for youngsters diagnosed with the cruel disease, but now gene therapy can potentially give babies the ability to sit, crawl and walk.

Zolgensma, which has a list price of £1.795 million per single dose, was made available on the NHS following a landmark deal struck with manufacturers Novartis Gene Therapies in March.

In studies, a single treatment with Zolgensma has helped babies with SMA to sit, crawl and walk and also prevented them from having to be put on a ventilator.

Four specialist NHS centres, Manchester University NHS Foundation Trust, Sheffield Children’s NHS Foundation Trust, University Hospitals Bristol and Weston NHS Foundation Trust and Evelina London Children’s Hospital, have now been commissioned across England to administer the treatment.

Sir Simon Stevens, NHS chief executive, said: “It is fantastic news that this revolutionary treatment is now available for babies and children like Arthur on the NHS. The NHS Long Term Plan committed to securing cutting edge treatments for patients at a price that is fair to taxpayers. Zolgensma is the latest example of the life-changing therapies that the NHS is now routinely using to transform the lives of patients and their families.”