Science

NHS secures world's most expensive drug to treat babies with rare muscle disease

Patients will be able to access Zolgensma, a one-time gene therapy, said NHS England.

Up to 80 babies a year born with a rare muscle-wasting disease could receive life-changing treatment after the most expensive drug in the world was licensed for use on the NHS in England.

Infants born with Spinal Muscular Atrophy (SMA), an often fatal condition which affects the spinal cord nerves causing paralysis, will be able to access Zolgensma, a one-time gene therapy, said NHS England.

The drug, which replicates a missing gene and restores nerve and muscle function, has a reported list price of nearly £1.8 million per dose.

But NHS England said it had struck a “landmark confidential deal” ensuring patients can get the crucial treatment “at a price that is fair to taxpayers”.

NHS England chief executive Sir Simon 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.”

Babies born with severe type 1 SMA – the most common form of the condition – have a life expectancy of just two years.

But studies show a one-time intravenous infusion of Zolgensma can help infants breathe without ventilators, sit up without help, and crawl and walk.

As many as 80 babies and young children could benefit from the life-changing treatment each year, said NHS England.

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

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.

“Every child deserves to enjoy a happy, healthy future, free from pain, and the NHS is working tirelessly to make this happen.”

The deal was secured with US-based manufacturer Novartis Gene Therapies.

The National Institute for Health and Care Excellence (Nice) published draft guidance recommending treatment with Zolgensma for babies aged up to 12 months with type 1 SMA.

A Nice statement said: “Despite the high cost of the treatment it can be recommended for use on the NHS because of the evidence of exceptional benefit to young babies, potentially allowing them to reach normal childhood developmental milestones.

“Because of the limited trial data for children aged seven to 12 months, their treatment should be discussed by a national multidisciplinary clinical team.”

Sir Simon added: “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.”

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