The NHS will offer the ‘world’s most expensive’ drug at £1.8m per dose to treat the baby spine condition that typically kills infants before they are two years old
- Around 65 babies born each year in England suffer from spinal muscular atrophy
- The causes of the disease cause muscle weakness, paralysis and respiratory problems
Babies born with a genetic condition that leads to a life expectancy of just two years without intervention will now be cured on the NHS with a new £1.8million drug.
Infants with severe spinal muscular atrophy (SMA), which causes muscle weakness and leads to paralysis and breathing problems, will now be able to take the gene therapy drug Zolgensma.
Treatment Observatory The National Institute for Health and Care Excellence (Nice) has authorized the use of onasemnogene abeparvovec, more commonly known by its brand name.
About 65 babies born each year in England suffer from spinal muscular atrophy, which is the leading genetic cause of death in children.
The disease causes a defect in a gene called SMN1, which makes essential proteins and thus allows spinal nerves in the spinal cord to control muscle movement.
Arthur Morgan, then five months old, with his father Reece Morgan in June 2021 after becoming the first patient in England to undergo gene therapy using Zolgensma
The most severe form of the disease, SMA type 1, can develop in infants under six months old – with a 90% chance of dying before the age of two if left untreated. The temperature reports.
But Zolgensma has been shown in studies to help sufferers sit, crawl and walk – things they normally could never do – and also prevent them from being put on a ventilator.
The single infusion, administered over just one hour, uses a harmless virus to deliver a healthy version of the SMN1 gene.
It passes into nerve cells to replace the faulty gene and restores normal function, allowing the baby to live a healthy life with minimal symptoms.
It comes after one-year-old Edward Willis-Hall was given the drug and then took his first assisted steps in January earlier this year.
His mother Megan Willis, 30, has spoken of her joy after her son took steps she never thought possible.
He can roll over, sit up, stand for five minutes in gaiters that support his legs, and take steps in a harness.
She told the BBC: ‘We are so proud of Edward. He’s doing incredibly well. It far exceeds our expectations. We are incredibly lucky.
Ms Willis, who was told her son had spinal muscular atrophy when he was seven weeks old, has set a goal for him to sit up unaided. She said: “All I ever wanted was for him to be able to sit down and I knew he would have an amazing life then.”
Nice said purchasing the drug was a “cost-effective” use of resources.
Currently, only babies who have a family history of SMA will be tested.
The single infusion, administered over just one hour, uses a harmless virus to deliver a healthy version of the SMN1 gene
But campaigners are pushing for all five-day-old infants to undergo checks diagnosing nine conditions.
Laurent Servais, Professor of Pediatric Neuromuscular Diseases at the University of Oxford, said: “I have seen too many families devastated by this disease, but now we have treatment options.
“However, waiting for symptoms to appear is still too late. Every day we delay finding and treating these infants, we could be responsible for a child spending their life in a wheelchair.
“There is 100 per cent consensus among UK experts, among global experts, among patients: we need to screen to save lives, we need to screen to save quality of life and we need to screen to save money. “