Breakthrough gene-therapy drug used by NHS for the first time

The first patient has been treated with Zolgensma – a breakthrough gene therapy – for patients with Spinal Muscular Atrophy (SMA).

Previously no treatment was available for SMA, but now gene-therapy offers a way forward for families.

The gene therapy drug produced by Novartis Gene Therapies, was procured in March, following a landmark deal secured by NHS England. Zolgensma, has a list price of £1.795 million per single dose, and we reported back in March that the NHS had struck a deal with the pharmaceutical giants.

SMA gives babies born with the disease a life expectancy of just two years, in studies conducted so far, Zolgensma helped babies sit, crawl, and walk and prevented them using a ventilator.

NHS chief executive Sir Simon Stevens 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.”

Dr Elizabeth Wraige, Consultant Paediatric Neurologist at Evelina London Children’s Hospital, added: “We are very excited to be able to provide this life-changing treatment to infants with the severe form of Spinal Muscular Atrophy at Evelina London Children’s Hospital.

“Babies like Arthur, who is first to receive this treatment in England, may now be able to have a one-off gene therapy to treat the root cause of SMA with the aim of preventing the further progression or even onset of this debilitating condition. This treatment will bring hope to families affected by SMA who have fought so courageously against it. ”

Four NHS sites were commissioned to use the breakthrough treatment and have been announced as 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.

Evelina London Children’s Hospital will collaborate with Great Ormond Street Hospital for Children NHS Foundation Trust on the use of the drug.