Kate Cardente has her then-daughter Ainsley, three months old, while undergoing gene therapy for spinal muscular atrophy (SMA). SMA is a disease that is the leading genetic cause of death in babies. Ainsley receives a unique infusion of Zolgensma.
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LONDON – A drug that has been labeled the “most expensive drug in the world” has been approved by the UK’s National Health Service, a measure that could change the lives of babies and children with a rare genetic disorder.
Innovative gene therapy called “Zolgensma” is not cheap, with a list price of £ 1.79 million ($ 2.48 million) per dose, NHS England said in a statement on Monday.
The drug will be used for infants and young children suffering from spinal muscular atrophy, a rare and often fatal genetic disease that causes paralysis, muscle weakness, and progressive loss of movement.
Babies born with severe type 1 SMA, the most common form of the condition, have a life expectancy of just two years.
The drug will be available from the UK Health Service, which provides free medical care at the time of delivery, “at a price that is fair to taxpayers following a flagship confidential deal reached by the NHS England,” it announced Monday. executive, Simon Stevens. NHS England did not disclose the price paid.
The NHH is funded primarily by the government through general taxes, so the drugs and treatments it approves and uses must go through a rigorous analysis of their profitability.
Zolgensma, manufactured by Novartis Gene Therapies (part of the US pharmaceutical company Novartis), has been shown in studies to help babies achieve milestones such as breathing without a fan, sitting alone and crawling and walking after a single infusion. treatment.
The latest data suggest that Zolgensma can provide rapid and sustained improvement in motor function in young children with type 1 SMA and prolong their life.
Up to 80 babies and young children a year could benefit from gene therapy, according to the NHS.
Life changer
Stevens said the deal was a “life change for young people with this cruel disease and for their families.”
“Spinal muscle 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, successfully negotiating behind the scenes to ensure a fair price for taxpayers.” .
He said the deal showed that while the health service remained under pressure due to the Covid-19 pandemic, the NHS still “cared for millions of other patients, for whom real medical advances are now possible.”
The agreement with Novartis Gene Therapies guarantees the drug for NHS patients in England with a substantial confidential discount and paves the way for the National Institute for Excellence in Health and Care (NICE), the public body which issues guidelines on the cost-effectiveness of medicines and treatments used by the NHS – to publish draft guidelines recommending treatment with Zolgensma.
The terms of the agreement mean that some young children who do not currently meet the NICE recommendation criteria will also be eligible to be treated by a national clinical team made up of the country’s leading experts in the treatment of SMA.
The NHS said it is ready to accelerate the introduction of highly complex and innovative gene therapy and will not wait until NICE publishes the final guidelines to begin. This approach is supported by NICE given the importance of administering timely treatment as soon as possible, he added.
Drug approval marks the second medical treatment available now for young people with SMA. He added that future treatment possibilities also look promising, as NICE is reviewing another SMA drug.