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NHS England agrees deal for gene therapy for spinal muscular atrophy

BMJ 2021; 372 doi: (Published 08 March 2021) Cite this as: BMJ 2021;372:n653
  1. Elisabeth Mahase
  1. The BMJ

NHS England has agreed a deal to ensure that Zolgensma (onasemnogene abeparvovec), a one-off gene therapy medicine, will be made available for patients with spinal muscular atrophy.

The drug, labelled the most expensive in the world at £1.79m a dose (€2.09m; $2.48m), will be sold to the NHS at a discounted rate, although the details of the deal are confidential. The move means that as many as 80 babies and young children a year could be given the life changing treatment.

Spinal muscular atrophy is a rare and often fatal genetic disease that causes paralysis, muscle weakness, and progressive loss of movement. However, a single intravenous infusion of Zolgensma, containing a replica of the missing gene SMN1, has been shown to help babies breathe without a ventilator, sit up on their own, and crawl and walk.

The drug’s manufacturer, Novartis, has also said that the latest data show that treatment could also prolong life. Babies born with severe type 1 spinal muscular atrophy, the most common form, have a life expectancy of 2 years.

NHS England said that it would fast track the gene therapy rather than waiting until the National Institute for Health and Care Excellence published its final guidance, an approach supported by the institute because of the importance of administering the treatment as early as possible.

Novartis was previously criticised after it decided to run a lottery programme to choose four babies to receive the gene therapy. The lottery drew condemnation from a patient group, SMA Europe, which said that the scheme would make babies compete against each other for a lifesaving treatment and could split tightly knit communities.1


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