The NHS has treated the first patient with Zolgensma, Novartis’ spinal muscular atrophy (SMA) gene therapy.
Five-month-old Arthur Morgan, who was diagnosed with SMA in May, has received the once-off gene therapy at Evelina London Children’s Hospital.
SMA is a rare genetic disease that causes paralysis, muscle weakness and progressive loss of movement. Children born with SMA type 1 typically have a life expectancy of around two years.
The list price for Zolgensma (onasemnogene abeparvovec) is £1.79m per dose, and has previously been labelled as the ‘most expensive drug in the world’.
The first administration of Zolgensma comes only a few months after Novartis and NHS England reached a landmark deal for the gene therapy.
NHS England said that the gene therapy will be available to patients at a ‘price that is fair to taxpayers’, adding that the deal secured the treatment at a substantial discount, although this has been kept confidential.
Following the deal, the National Institute for Health and Care Excellence (NICE) published a draft recommendation for the gene therapy.
This recommendation advises the use of Zolgensma on the NHS for babies aged up to 12 months with SMA type 1 before they develop symptoms.
NICE has agreed a managed access agreement for Zolgensma, meaning that further data is set to be collected prior to the publication of the final guidance. According to NHS England, up to 80 patients will be able to benefit from treatment with Zolgensma each year.
The gene therapy will also be offered to young children with SMA type 1 who do not meet the recommendation criteria. The use of the potential gene therapy treatment for these patients will be discussed by a national multidisciplinary team (MDT).
“It is fantastic news that this revolutionary treatment is now available for babies and children like Arthur on the NHS. The NHS Long Term Plan committed to securing cutting edge treatments for patients at a price that is fair to taxpayers,” said Simon Stevens, chief executive of the NHS.
“Zolgensma is the latest example of the life-changing therapies that the NHS is now routinely using to transform the lives of patients and their families,” he added.
The four specialist NHS sites that have been commissioned to administer Zolgensma across England are: Manchester University NHS Foundation Trust, Sheffield Children’s NHS Foundation Trust, University Hospitals Bristol and Weston NHS Foundation Trust and Evelina London Children’s Hospital.