Baby becomes first NHS patient to be treated with world's most expensive drug

1 June 2021, 06:44

Arthur Morgan, five-months, has become the first NHS patient to be treated with the world's most expensive drug
Arthur Morgan, five-months, has become the first NHS patient to be treated with the world's most expensive drug. Picture: PA

By Kate Buck

A five-month-old baby has become the first patient in England treated with a potentially life-saving drug on the NHS that can prolong the lives of children with spinal muscular atrophy.

Arthur Morgan, who was diagnosed with the condition earlier this month, received the one-off gene therapy at Evelina London Children's Hospital on May 25.

Until two years ago, there were no treatment options available for children with spinal muscular atrophy (SMA), which is the leading genetic cause of death for children.

But babies could potentially have the ability to sit, crawl and walk after being treated with US gene therapy Zolgensma, which has been called the most expensive drug in the world.

Zolgensma, which has a list price of £1.79 million per dose, was made available on the NHS after the health service struck a deal with manufacturers Novartis Gene Therapies in March.

Baby Arthur pictured with his dad Reece Morgan
Baby Arthur pictured with his dad Reece Morgan. Picture: PA

Baby Arthur, who was born six weeks premature in December, underwent the gene therapy infusion last week after being diagnosed with SMA less than three weeks earlier.

His father Reece Morgan, 31, who works as a self-employed plasterer, said: "When we found out that Arthur would get the treatment, and be the first patient, I just broke down.

"It had been such a whirlwind few weeks, filled with lots of anxiety and adjustment, as we learnt about his condition and what it might mean for him and our family.

"We still don't know what the future will hold, but this gives Arthur the best possible chance to give him the best possible future."

Babies born with Type 1 SMA, which is the most common form of the condition, experience progressive muscle weakness, loss of movement, difficulty breathing, and have a life expectancy of just two years.

Studies found that 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
Studies found that 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. Picture: PA

Studies found that 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.

Four specialist NHS centres have now been commissioned across the country to administer the treatment, including Evelina London Children's Hospital, where Arthur was treated.

The other sites are Manchester University NHS Foundation Trust, Sheffield Children's NHS Foundation Trust and University Hospitals Bristol and Weston NHS Foundation Trust.

Dr Elizabeth Wraige, consultant paediatric neurologist at Evelina London Children's Hospital, said: "This treatment will bring hope to families affected by SMA who have fought so courageously against it."

NHS chief executive Sir Simon Stevens added: "It is fantastic news that this revolutionary treatment is now available for babies and children like Arthur on the NHS.

"The NHS Long Term Plan committed to securing cutting edge treatments for patients at a price that is fair to taxpayers.

"Zolgensma is the latest example of the life-changing therapies that the NHS is now routinely using to transform the lives of patients and their families."

Health Secretary Matt Hancock said: "I am so glad young Arthur can access this potentially life-changing treatment on the NHS. I hope it grants his family and many others renewed hope that more children's lives can be transformed."

More Latest News

See more More Latest News

Artist's impression of the new national biosecurity centre at Weybridge, Surrey

New centre to tackle 'risk of future pandemics' gets billion-pound investment

Screenings for cervical cancer save at least 2,000 lives each year in the UK

NHS to offer at-home cervical cancer screening test kits that 'could save 5,000 lives per year'

Zelensky visits 10 Downing Street for talks with Keir Starmer on Monday

Zelensky warns Putin could launch an attack on NATO 'within five years'

Tom Cruise and Brad Pitt arriving at the F1 The Movie premiere in Leicester Square.

'Interview with the Vampire' co-stars Brad Pitt and Tom Cruise make 'surprise' public reunion after 24 years

The first Brits are arriving in the UK after being evacuated from Israel.

First Brits rescued from Israel touch down at Heathrow Airport as more to arrive in coming days

The president of the Royal College of Psychiatrists (RCPsych) said professionals are in distress

Stretched mental health services leaving psychiatrists ‘in emotional and ethical distress’

Jobs will be created at warehouses in locations including Hull and Northampton

Amazon’s new UK warehouses to hire thousands as Starmer hails ‘win’ for Britain

Iran has launched an attack on a US military base.

Trump claims Israel and Iran have agreed 'complete and total ceasefire' in wake of Iranian strike on US air base

A Qatar Airways flight from Manchester was forced to divert earlier this evening

UK flight bound for Doha forced to turn back after Iran launches missiles at US air base in Qatar

Sir Keir Starmer will fly to the Hague today for crunch meetings against a backdrop of global volatility in the Middle East and Ukraine.

UK and NATO allies to include 'money spent on tackling small boats' to boost defence spending in bid to appease Trump

The

Thousands raised after family die in motorway crash - daughter, 8, is only survivor

Kate Shemirani, conspiracy theorist, anti-vaxxer and former nurse

Daughter of notorious conspiracy theorist died of cancer after 'falling for mother's theories', her brothers say

Emma Raducanu during a practice session during Eastbourne Open, where she is currently competing

Raducanu says Wimbledon 'did amazing job' after stalker tried to buy match tickets

Large fire engulfs the abandoned Hotspur Press mill building near Oxford Road in Central Manchester. Credit Milo Chandler/Alamy Live News

Major fire in Manchester city centre causes serious disruption

Notorious criminal John 'Goldfinger' Palmer was murdered at his home in Brentwood in Essex in 2015. His killer has never been found

Who killed John 'Goldfinger' Palmer? Police make fresh appeal 10 years after his death

An RAF flight has left Israel carrying 63 UK nationals and their dependents, as the UK government begins evacuating British citizens from the country amid ongoing attacks between Iran and Israel.

First RAF flight evacuates 63 Britons from Israel as Lammy confirms one UK national injured in Iran missile attacks