Up to 80 babies a year born with a rare muscle wasting disease could receive life-changing treatment after the world’s most expensive drug was approved for use on the NHS in England.
Babies born with Muscular Spinal Atrophy (SMA), a often fatal condition that affects spinal nerves causing paralysis, will be unable to access Zolgensma, a one-time gene gene, NHS England said.
The drug, which reproduces a missing gene and restores zero activity and muscle, is priced at a reported list price of nearly £ 1.8 million per dose.
But NHS England said it had struck a “confidentiality deal” ensuring patients receive the essential treatment “at a fair price for taxpayers”.
NHS England chief executive Sir Simon Stevens said: “This agreement is a way of life for young people with this serious illness and for their families.
“Muscular Spinal Atrophy is the leading genetic cause of death in infants and young children, which is why NHS England has moved mountains to provide this treatment, successfully negotiating hard behind the scenes to ensure a fair price for taxpayers. ”
Babies born with type 1 SMA – the most common form of the condition – have a life expectancy of just two years.
But studies show that one-time intravenous infusion of Zolgensma can help babies breathe without cooling, sit up unaided, and crawl and walk.
Up to 80 babies and young children could benefit from the life-changing treatment each year, NHS England said.
The drug contains a replica of the missing SMN1 gene and its active ingredient, onasemnogene abeparvovec, enters the nerves and regenerates the gene, which produces needed proteins for zero activity and muscle movement control.
Health Secretary Matt Hancock said: “This drug is a play-off for babies born with this musculoskeletal disorder and I am delighted that the NHS will be able to offer this treatment to babies and young people. young children soon.
“Every child deserves a happy, healthy, pain-free future, and the NHS is working hard to make this happen.”
The agreement was secured by US-based manufacturer Novartis Gene Therapies.
The National Institute for Health and Care Excellence (Nice) has published a draft guideline recommending treatment with Zolgensma for babies up to 12 months with type 1 SMA.
The Nice statement said: “Despite the high cost of the treatment it can be recommended for use on the NHS because of evidence of exceptional benefit to young babies, possibly enabling them to reach normal childhood developmental milestones.
“Given the limited trial data for children aged seven to 12 months, their treatment should be discussed with a national multidisciplinary clinical team.”
Sir Simon said: “While the health service remains under significant pressure from Covid, and NHS England is also focused on leading the rollout of Covid’s national vaccine, today’s agreement is an important reminder that the NHS looking after millions of other patients as well, for it is now possible to make medical progress. “