NHS England strikes deal on life-saving gene-therapy drug that can help babies with rare genetic disease move and walk
A life-saving drug that can enable mobility in babies and young children suffering from a rare genetic condition will be available on the NHS, chief executive Sir Simon Stevens yesterday announced.
Zolgensma, which has a reported list price of £1.79 million per dose and is labelled the most expensive drug in the world, will be available to patients at a price that is fair to taxpayers after a landmark confidential deal struck by NHS England.
The one-off gene therapy treats Spinal Muscular Atrophy (SMA), 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.
In studies Zolgensma, manufactured by Novartis Gene Therapies, has helped babies to reach milestones such as breathe without a ventilator, sit up on their own and crawl and walk after a single infusion treatment.
NHS England Chief Executive Sir Simon Stevens yesterday said:
“This deal is a life-changer for youngsters with this cruel disease and for their families.
“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.
“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.”
The deal struck with Novartis Gene Therapies, secures the drug for NHS patients in England at a substantial confidential discount and paves the way for the National Institute for Health and Care Excellence (NICE) to publish draft guidance recommending treatment with Zolgensma.
The terms of the deal mean that some young children that currently fall outside the NICE recommendation criteria will also be eligible to be considered for treatment by a national multidisciplinary clinical team (MDT) made up of the country’s leading experts in the treatment of SMA.
This means as many as 80 babies and young children could potentially benefit from the life-changing gene therapy a year.
The treatment is given as a single intravenous infusion and contains a replica of the missing gene SMN1.
The active ingredient onasemnogene abeparvovec passes into the nerves and restores the gene, which then produces proteins essential for nerve function and controlling muscle movement.
The latest data suggests that Zolgensma can provide rapid and sustained improvement in motor function for young children with type 1 SMA and prolong their lives.
The NHS is ready to fast-track the introduction of the highly complex and innovative gene therapy and will not wait until NICE publish final guidance to get going. This approach is backed by NICE given the importance of administering the one-off treatment as early as possible.
The NHS is currently undertaking a rigorous process to identify centres of excellence to provide the full range services required to administer the treatment safely, with new specialist services treating patients in the coming months. These services require paediatric intensive care and a wide range of other clinical expertise such as neuromuscular, paediatric respiratory medicine, immunology, infectious diseases, hepatology, renal, cardiology and endocrinology.
The approval of the drug marks the second medical treatment now available for youngsters with SMA, after Spinraza became available on the NHS to eligible patients in May 2019. Future treatment possibilities also look promising, with another SMA drug currently being reviewed by NICE.
This is the latest in a series of ‘smart deals’ the NHS has agreed to secure innovative medicines for patients over the past year, while also treating thousands of people with COVID-19 and delivering a rapid COVID-19 vaccines rollout vaccinating 18 million people. Recent agreements have included a first full-access deal in Europe for CAR-T therapy, KTE-X19 for mantle-cell lymphoma, and siponimod as a first oral treatment for secondary progressive multiple sclerosis.
Latest News from
NHS retention drive expanded across the country with thousands fewer staff leaving frontline roles07/12/2023 15:15:00
Thousands fewer staff left the NHS last year, thanks to a major new retention programme which is now being expanded across the country.
Identified safety risks with the Euroking maternity information system07/12/2023 14:15:00
A National Patient Safety Alert has been issued by the NHS England National Patient Safety Team on identified safety risks with the Euroking maternity information system.
Top A&E doctors warn of rising pressure from winter viruses07/12/2023 13:15:00
Frontline doctors are warning winter virus cases are “creeping up” alongside pressure on emergency departments, with new NHS data showing the number of flu patients in hospital is up more than half in a week.
NHS online GP registration service rolled out to over 2,000 practices07/12/2023 12:15:00
An NHS pledge to roll out a new online GP registration service to 2,000 GP practices by the end of the year has been achieved ahead of schedule.
Millions more people given access to their GP records online05/12/2023 09:15:00
More than four in five GP practices [81.1%] in England are now giving patients access to their new health records online, thanks to the NHS primary care access recovery plan.
NHS urges people to get winter jabs with two weeks until online bookings close04/12/2023 10:25:00
With just two weeks until online bookings for COVID and flu vaccines close, the NHS is urging anyone still eligible for these vaccines to book an appointment and get potentially lifesaving protection ahead of Christmas.
NHS to offer stem cell transplants to cure life-limiting blood disorder30/11/2023 16:20:00
Patients with severe inherited blood disorders will be offered stem cell transplants on the NHS that could cure their condition and help avoid life-long blood transfusions.
Hundreds of patients in hospital with norovirus ahead of winter30/11/2023 14:15:00
The number of patients in hospital with norovirus last week was almost triple the number during the same period last winter, new NHS figures show today.