Five-month year old Arthur Morgan has become the first patient on the NHS to receive the world’s most expensive drug.
Gene therapy
Arthur Morgan was diagnosed with spinal muscular atrophy (SMA) in May 2021.It is a condition which is the leading genetic cause of death for children and which there was no treatment for until around two years ago.
SMA is a rare, inherited, neuromuscular disease that affects around one in every 10,000 births. It’s caused by the lack of a functional SMN1 gene, resulting in the loss of muscle functions, including breathing and basic movement. If untreated, SMA Type 1 can lead to death or the permanent need for ventilation by the age of two.
The gene therapy Zolgensma was developed by AveXis and acquired through Novartis to treat SMA. It was made available on the NHS through a draft recommendation from the National Institute of Health and Clinical Excellence (NICE).
Zolgensma works by replacing the function of the faulty SMN1 gene. The gene therapy is not designed as a cure however, but rather to halt the progression of the disease. A small-scale clinical trial examining the long-term efficacy of the drug showed that Zolgensma was able to help infants avoid the need for ventilation and be able to walk, sit up, and maintain motor function’s.
A single dose of the gene therapy costs £1.79 million.
Arthur Morgan received the one-off gene therapy at Evelina London Children’s Hospital on 25 May.
His father, Reece Morgan, said: “This is the best possible treatment and will give him the best life he can have, and we are so grateful that the NHS is here for him.”
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.”
Four specialist NHS centres have now been commissioned across England to administer the treatment.
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.”
Cliodhna McDonough, life sciences regulatory lawyer at Fieldfisher says that the news should be welcome to pharmaceutical companies operating in the gene therapy space.
“Overall, this is exciting news for the future of medicine. It's good to see that pharmaceutical companies are open to negotiating prices to get treatments to patients. Moreover, the use of another treatment by the NHS, is a clear signal to the UK life sciences industry, particularly those operating within the cell and gene therapy space that the pathway to market for innovative treatments is getting clearer,” McDonough said.