NHS treats first patient with the ‘world’s most expensive drug’
A five-month old baby has become the first patient to receive a potentially life-saving drug on the NHS that can prevent paralysis and prolong the lives of children with Spinal Muscular Atrophy (SMA).
Arthur Morgan, who was diagnosed with SMA earlier this month, received the one-off gene therapy at Evelina London Children’s Hospital on May 25.
Dad Reece Morgan, 31, yesterday said:
“This is the best possible treatment and will give him the best life he can have, and we are so grateful that the NHS is here for him.”
Untreated SMA is the leading genetic cause of death for children, and until two years ago there were no treatment options available for youngsters diagnosed with the cruel disease, but now gene therapy can potentially give babies the ability to sit, crawl and walk.
The NHS Long Term Plan committed to using cutting edge treatments and therapies to save and improve patients’ lives.
Zolgensma, which has a list price of £1.795 million per single dose, was made available on the NHS following a landmark deal struck with manufacturers Novartis Gene Therapies in March.
NHS chief executive Sir Simon Stevens yesterday said:
“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.”
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.
Reece and Arthur’s mum Rosie, from south London, had taken him to their local A&E department after they noticed that he was immobile, floppy and could no longer hold up his head.
Doctors suspected that he had SMA and carried out genetic tests to confirm the diagnosis before transferring him to Evelina London Children’s Hospital, part of Guy’s and St Thomas’ NHS Foundation Trust.
Reece, who works as a self-employed plasterer, yesterday 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.
“The way the treatment works is incredible, where the gene is hidden in a virus, and is given to him in a simple canula. 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 – 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.
In studies, 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 England to administer the treatment.
The four sites now confirmed are Manchester University NHS Foundation Trust, Sheffield Children’s NHS Foundation Trust, University Hospitals Bristol and Weston NHS Foundation Trust and Evelina London Children’s Hospital (part of Guy’s and St Thomas’ NHS Foundation Trust), with agreement for Evelina London Children’s Hospital to develop a collaborative model with Great Ormond Street Hospital for Children NHS Foundation Trust.
Dr Elizabeth Wraige, Consultant Paediatric Neurologist at Evelina London Children’s Hospital yesterday said:
“We are very excited to be able to provide this life-changing treatment to infants with the severe form of Spinal Muscular Atrophy at Evelina London Children’s Hospital.
“Babies like Arthur, who is first to receive this treatment in England, may now be able to have a one-off gene therapy to treat the root cause of SMA with the aim of preventing the further progression or even onset of this debilitating condition. This treatment will bring hope to families affected by SMA who have fought so courageously against it. ”
Sally-Anne Tsangarides, General Manager at Novartis Gene Therapies in the UK yesterday said:
“Novartis is delighted to be part of this remarkable milestone – it is a hugely important step for babies in England diagnosed with this devastating genetic condition and we thank all those who have been involved in the landmark agreement that has made it possible.”
Liz Ryburn Support Team Manager at SMA UK yesterday said:
“It’s very heartening to see the first child being treated via the NHS in England with this ground-breaking gene therapy and we know there has been a huge amount of work going on by clinicians and NHS England to get this programme up and running.
“Zolgensma could be potentially lifechanging for some babies and children diagnosed with SMA, and as we support families through this understandably anxious period, we hope those currently waiting will soon be able to have full discussions with their clinical team to see if this treatment option will also be appropriate for their child.”
Zolgensma is the latest in a series of ‘smart deals’ the NHS has agreed to secure innovative medicines for patients over the past year, while also treating thousands of people with COVID-19 and delivering a rapid COVID-19 vaccines rollout vaccinating 32 million people.
Recent agreements have included a first full-access deal in Europe for CAR-T therapy, KTE-X19 for mantle-cell lymphoma, and siponimod as a first oral treatment for secondary progressive multiple sclerosis.
Health and Social Care Secretary Matt Hancock yesterday 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.
“Through the NHS Long Term Plan, we are doing everything we can to provide people with access to the latest generation of innovative medicines though the NHS.
“Arthur’s story is the latest example of our brilliant life sciences industry working hand in hand with the NHS to put patients first.”
Latest News from
NHS England proposes new mental health access standards22/07/2021 12:20:00
The NHS is set to take another major step towards improving patient access to mental health services with the introduction of five new waiting time guarantees, under plans set out today.
NHS England announces new Innovative Medicines Fund to fast-track promising new drugs21/07/2021 12:20:00
NHS patients are set to benefit from early access to potentially life-saving new medicines, including cutting-edge gene therapies, thanks to a new Innovative Medicines Fund and £680 million of ringfenced funding, the NHS chief executive has announced today.
NHS workforce more diverse than any point in its history, as health service commits to more action on representation20/07/2021 16:10:00
A first of its kind report looking into race equality among England’s doctors has found that the number from black and ethnic minority backgrounds working for the NHS is the highest on record.
NHS ‘Grab A Jab’ Weekend In Final Drive To Get The Country Protected16/07/2021 16:43:00
People can ‘grab-a-jab’ when they pop into Primark or while sunbathing in the park as the NHS vaccination programme seeks to maximise protection before Covid restrictions are lifted.
NHS Patients, staff and visitors must continue to wear face coverings in healthcare settings16/07/2021 13:48:00
England’s Chief Nurse yesterday reminded the public that everyone accessing or visiting healthcare settings must continue to wear a face covering and follow social distancing rules.
NHS text message drive to encourage millions to bring forward second vaccine16/07/2021 09:15:00
Hundreds of thousands of text messages will be sent by the NHS COVID-19 Vaccination programme this week reminding people to bring forward their second dose appointment.
NHS investment boost to ambulance staff numbers ahead of winter15/07/2021 16:10:00
Ambulance trusts in England will be given an extra £55 million to boost staff numbers ahead of winter, the NHS recently (14 July 2021) announced.
Inappropriate anticoagulation of patients with a mechanical heart valve15/07/2021 09:15:00
A National Patient Safety Alert has been issued on the risk of inappropriate anticoagulation of patients with a mechanical heart valve.