Babies with rare muscle disease can now get 'life-changer' drug on the NHS
Patients with spinal muscular atrophy will be able to access Zolgensma, a one-time gene therapy, said NHS England.
Up to 80 babies a year are born with a rare muscle-wasting disease – and now they could receive life-changing treatment, after the most expensive drug in the world was licensed for use on the NHS in England.
Infants born with spinal muscular atrophy (SMA), an often fatal condition which affects the spinal cord nerves causing paralysis, will be able to access Zolgensma, a one-time gene therapy, said NHS England. The drug, which replicates a missing gene and restores nerve and muscle function, has a reported list price of nearly £1.8m per dose.
But NHS England said it had struck a “landmark confidential deal” with US-based manufacturer Novartis Gene Therapies, ensuring patients can get the crucial treatment “at a price that is fair to taxpayers”.
“This deal is a life-changer for youngsters with this cruel disease and for their families," said NHS England chief executive Sir Simon Stevens.
“Spinal muscular atrophy is the leading genetic cause of death among babies and young children, which is why NHS England has moved mountains to make this treatment available, while successfully negotiating hard behind the scenes to ensure a price that is fair to taxpayers.”
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Babies born with severe type 1 SMA – the most common form of the condition – have a life expectancy of just two years. But studies show a one-time intravenous infusion of Zolgensma can help infants breathe without ventilators, sit up without help, and crawl and walk.
As many as 80 babies and young children could benefit from the life-changing treatment each year, said NHS England.
The drug contains a replica of the missing gene SMN1 and the active ingredient, onasemnogene abeparvovec, enters the nerves and restores the gene, which then produces proteins needed for nerve function and controlling muscle movement.
The National Institute for Health and Care Excellence (NICE) published draft guidance recommending treatment with Zolgensma for babies aged up to 12 months with type 1 SMA.
The "exceptional benefit" to young babies justifies the high cost of the treatment, NICE said, as the drug potentially allows them to reach normal childhood developmental milestones.
“Although the health service is still under real pressure from COVID, and NHS England is also focused on leading the national COVID vaccination rollout, today’s agreement is an important reminder that the NHS is looking after millions of other patients too, for whom real medical advances are now possible,” Stevens added.
Why can’t we remember early life?
Asked by: Karen Evans, Leeds
Our inability to remember anything from before the age of three or four is referred to as infantile amnesia and it’s still fairly mysterious. We do know that infants can form long-term memories: chat to a three-year-old about past events and you’ll see for yourself.
In fact, one study showed that three-year-olds had a memory of an adult they’d met just once when they were aged one. But for some reason, likely related to the immaturity of infant memory processes, our earliest memories are lost by the time we are about seven years of age.
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