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 (SMA).
Arthur Morgan, who was diagnosed with SMA earlier this month, received the one-off gene therapy at Evelina London Children’s Hospital. 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.
Baby Arthur, who was born six weeks premature in December, underwent the gene therapy infusion last week after being diagnosed with SMA less than three weeks earlier.
Babies born with type 1 SMA – the most common form of the condition – experience progressive muscle weakness, loss of movement, difficulty breathing, and have a life expectancy of just two years. 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 have now been commissioned across England to administer the treatment.
The four sites now confirmed are 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 (part of Guy’s and St Thomas’ NHS Foundation Trust), with agreement for Evelina London Children’s Hospital to develop a collaborative model with Great Ormond Street Hospital for Children NHS Foundation Trust.
Zolgensma is the latest in a series of ‘smart deals’ the NHS has agreed to secure innovative medicines for patients over the past year. Recent agreements have included a first full-access deal in Europe for CAR-T therapy, KTE-X19 for mantle-cell lymphoma, and siponimod as a first oral treatment for secondary progressive multiple sclerosis.
Arthur’s Dad Reece said: “When we found out that Arthur would get the treatment, and be the first patient, I just broke down. It had been such a whirlwind few weeks, filled with lots of anxiety and adjustment as we learnt about his condition and what it might mean for him and our family.
“The way the treatment works is incredible, where the gene is hidden in a virus, and is given to him in a simple canula. We still don’t know what the future will hold, but this gives Arthur the best possible chance to give him the best possible future.”
Dr Elizabeth Wraige, Consultant Paediatric Neurologist at Evelina London Children’s Hospital said: “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.”
Sally-Anne Tsangarides, General Manager at Novartis Gene Therapies in the UK said: “Novartis is delighted to be part of this remarkable milestone – it is a hugely important step for babies in England diagnosed with this devastating genetic condition and we thank all those who have been involved in the landmark agreement that has made it possible.”