A 5-month old baby has become the first patient in England to receive a potentially life-saving drug on the NHS that could prevent paralysis and prolong the lives of children with spinal muscular atrophy (SMA).
The drug, onasemnogene abeparvovec (Zolgensma, Novartis Gene Therapies) was recommended for NHS use in England in March this year by the National Institute for Health and Care Excellence (NICE), and in Scotland by the Scottish Medicines Consortium, for babies aged up to 12 months with type 1 SMA.
At a list price of £1.795 million per single dose, Zolgensma would be the most expensive treatment ever approved by NICE. However, access to the drug came from a confidential commercial deal negotiated between NHS England and the manufacturer based on limited current evidence of efficacy.
Gene Therapy for Baby Arthur
Arthur Morgan, who was born 6 weeks premature in December, and diagnosed with SMA last month, received the one-off gene therapy at Evelina London Children's Hospital on May 25.
His parents, from south London, had taken him to A&E after they noticed that he was immobile, floppy, and could not hold his head up. Genetic testing confirmed a diagnosis of type 1 SMA. The condition is characterised by progressive muscle weakness, loss of movement, breathing difficulties, and a life expectancy of 2 years.
A single treatment with Zolgensma has helped babies with SMA to sit, crawl, and walk, and also prevented them from having to be put on a ventilator, NHS England said.
'Hope for Families'
Dr Elizabeth Wraige, consultant paediatric neurologist at Evelina London Children's Hospital said: "Babies like Arthur, who is first to receive this treatment in England, may now be able to have a one-off gene therapy to treat the root cause of SMA with the aim of preventing the further progression or even onset of this debilitating condition.
"This treatment will bring hope to families affected by SMA who have fought so courageously against it."
Arthur's father, Reece, said: "This is the best possible treatment and will give him the best life he can have, and we are so grateful that the NHS is here for him."
Following regulatory approval of Zolgensma, four specialist NHS centres were commissioned in England to administer the treatment. As well as Evelina London Children's Hospital, they are Manchester University NHS Foundation Trust, Sheffield Children's NHS Foundation Trust, and University Hospitals Bristol and Weston NHS Foundation Trust.
NHS Long Term Plan
The charity, Spinal Muscular Atrophy UK, said news of the treatment was "very heartening" and called for early diagnosis to enable treatment to stand the best chance of success.
Sir Simon Stevens, chief executive of NHS England, said: "The NHS Long Term Plan committed to securing cutting edge treatments for patients at a price that is fair to taxpayers."
Matt Hancock, England's Health Secretary, said that due to the Plan, "we are doing everything we can to provide people with access to the latest generation of innovative medicines though the NHS".
Around 65 babies are born each year in England with SMA, of whom about 60% are diagnosed with type 1 SMA, according to NICE.
Article image credit: PA Media
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Cite this: Peter Russell. Baby With Spinal Muscular Atrophy Treated With Zolgensma Gene Therapy - Medscape - Jun 01, 2021.