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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 cleared for use by the NHS in England.
Infants born with spinal muscular atrophy (SMA), an often fatal disease that affects nerves in the spinal cord causing paralysis, will be able to access Zolgensma, a one-time gene therapy, NHS England said.
The drug, which replicates a missing gene and restores nerve and muscle function, has a list price of nearly £ 1.8million per dose.
But NHS England said it had reached a “historic confidential agreement” ensuring that patients can receive crucial treatment “at a fair price to the taxpayer”.
NHS England Director General Sir Simon Stevens said: “This deal is changing the lives of young people with this cruel disease and their families.
‘Spinal muscular atrophy is the leading genetic cause of death in babies and young children, which is why NHS England has moved mountains to make this treatment available, while successfully negotiating behind the scenes to secure a price fair to taxpayers.
Babies born with severe type 1 ADS – the most common form of the disease – have a life expectancy of just two years.
But studies show that a single intravenous infusion of Zolgensma can help infants breathe without a ventilator, sit unaided, crawl and walk.
Up to 80 babies and toddlers could benefit from the life-changing treatment each year, NHS England said.
The medicine contains a replica of the missing SMN1 gene and the active ingredient, onasemnogene abeparvovec, enters the nerves and restores the gene, which then produces the proteins necessary for nerve function and the control of muscle movement.
Health Secretary Matt Hancock said: ‘This drug is a game changer for babies born with this rare muscle wasting disease and I am absolutely delighted that the NHS is soon able to offer this therapy to babies and young children.
“Every child deserves to enjoy a happy and healthy future without pain, and the NHS is working tirelessly to achieve this.”
The agreement was concluded with the American manufacturer Novartis Gene Therapies.
The National Institute for Excellence in Health and Care (Nice) has published a draft guideline recommending treatment with Zolgensma for babies under 12 months of age with type 1 SMA.
A statement from Nice said: ‘Despite the high cost of treatment, it may be recommended for use on the NHS due to evidence of exceptional benefit for young babies, potentially allowing them to reach developmental milestones. normal childhood.
“Due to limited trial data in children aged 7 to 12 months, their treatment should be discussed by a national multidisciplinary clinical team.”
Sir Simon added: ‘While the health service is still under real pressure from Covid and NHS England is also focused on leading the nationwide rollout of the Covid vaccination, the deal reached today is a reminder It is important that the NHS also take care of millions of other patients, because real medical breakthroughs are now possible.
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