Zolgensma: World’s most expensive drug licensed for use on NHS to treat Spinal Muscular Atrophy

The world’s most expensive drug has been licensed for use on the NHS in England.

Zolgensma has a reported list price of nearly £1.8m per dose, but the health service said it had struck a “landmark confidential deal” with its maker at a “price that is fair to taxpayers”.

The one-time gene therapy will be used to treat Spinal Muscular Atrophy (SMA) in infants. The often fatal muscle condition affects nerves in the spinal cord, leading to paralysis.

In the UK, up to 80 babies a year are born with SMA. Children who have the most common form of it – type 1 – have a life expectancy of only two years.

Zolgensma, which is made by Novartis Gene Therapies, replicates a missing gene and restores nerve and muscle function.

Studies have shown it can help infants to breathe without ventilators, sit up unaided, crawl and walk.

The National Institute for Health and Care Excellence (NICE) said there was “evidence of exceptional benefit to young babies, potentially allowing them to reach normal childhood developmental milestones”.

Sir Simon Stevens, chief executive of NHS England, said the deal is a “life-changer for youngsters with this cruel disease and for their families”.

He added: “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.”

The drug contains a replica of missing gene SMN1. Its active ingredient – onasemnogene abeparvovec – enters the nerves and restores the gene, which then produces proteins needed for nerve function and controlling muscle movement.

NICE has published draft guidance recommending treatment with Zolgensma for babies aged up to 12 months with type 1 SMA.

Health Secretary Matt Hancock said: “This drug is a game-changer for babies born with this rare muscle wasting disease and I am absolutely delighted the NHS will soon be able to offer this therapy to babies and young children.”

Sir Simon added: “Although the health service is still under real pressure from COVID, and NHS England is also focused on leading the national (coronavirus) 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.”

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