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 last month, received the one-off gene therapy at Evelina London Children’s Hospital on May 25.
The drug, Zolgensma, which has a list price of £1.795m per single dose, was made available on the NHS following a deal with manufacturers Novartis 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 experience progressive muscle weakness, loss of movement, difficulty breathing, with a life expectancy of just two years.
Health secretary Matt Hancock said: “I am so glad young Arthur can access this potentially life-changing treatment on the NHS. I hope it grants his family and many others renewed hope that more children’s lives can be transformed.
“Through the NHS Long Term Plan, we are doing everything we can to provide people with access to the latest generation of innovative medicines though the NHS.
“Arthur’s story is the latest example of our brilliant life sciences industry working hand in hand with the NHS to put patients first.”
Dr Elizabeth Wraige, consultant paediatric neurologist at Evelina London Children’s Hospital, said: “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 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.”