NHS England has revealed that 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.
Sir Simon Stevens said that 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 type 1 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.
Studies have found that Zolgensma has helped babies to reach milestones such as breathe without a ventilator, sit up on their own and move and walk after a single injection.
The National Institute for Health and Care Excellence (NICE) will publish draft guidance recommending treatment with Zolgensma.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.
Stevens 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.”
