Kate Cardente holds her then 3-month-old daughter, Ainsley, while undergoing gene therapy for spinal muscle atrophy (SMA). SMA is a disease that is the primary genetic cause of death in babies. Ainsley receives a one-time infusion from Z accordma.
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LONDON – A drug labeled the “most expensive drug in the world” has been approved by the UK’s National Health Service, a move that could change the lives of babies and children with a rare genetic condition.
The innovative gene therapy called “Z Accordingma” isn’t cheap, with a reported list price of £ 1.79 million ($ 2.48 million) per dose, NHS England said in a statement Monday.
The drug will be used for babies and young children suffering from spinal muscle 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 only two years.
The drug will be available from the UK health service, which provides free medical care at the point of delivery, “at a price that is reasonable to taxpayers following a historic NHS England confidential deal,” Chief Executive Simon Stevens announced Monday. . NHS England has not disclosed the price paid.
The NHS is primarily government-funded from general taxes, which is why the drugs and treatments it approves and uses must undergo a thorough analysis for their cost-effectiveness.
Manufactured by Novartis Gene Therapies (which is part of the US pharmaceutical company Novartis), Z accordma has been shown in studies to help infants reach milestones such as breathing without a ventilator, sitting upright on their own and crawling and walking after a single infusion. therapy.
The latest data suggests that Z Zalma may provide rapid and sustained motor improvement in young children with type 1 SMA and extend their lives.
As many as 80 babies and young children per year could potentially benefit from the gene therapy, according to the NHS.
Life changer
Stevens said the deal was a “life changer for young people with this cruel disease and for their families.”
“Spinal muscle atrophy is the leading genetic cause of death in infants and young children. That’s why NHS England has moved mountains to make this treatment available, while successfully negotiating behind the scenes to ensure a price that is fair to the taxpayer. “
He said the agreement showed that while the health service remained under pressure as a result of the Covid-19 pandemic, the NHS was still “caring for millions of other patients for whom real medical progress is now possible.”
The deal with Novartis Gene Therapies insures the drug for NHS patients in England at a significant confidential discount and paves the way for the National Institute for Health and Care Excellence (NICE) – the public body that provides guidance on the cost-effectiveness of drugs and treatments used by the NHS – to publish design guidelines recommending treatment with Z accordma.
The terms of the deal mean that some young children who currently fall outside the NICE recommendation criteria will also be eligible for treatment by a national clinical team made up of the country’s foremost experts on SMA treatment.
The NHS said it is poised to accelerate the introduction of the highly complex and innovative gene therapy and will not wait for NICE to publish final guidelines to get started. This approach is supported by NICE, given the importance of administering the one-time treatment as early as possible, it added.
The drug’s approval marks the second medical treatment now available for young people with SMA. It added that future treatment options also look very promising, with another SMA drug currently under review by NICE.