Parents ‘overwhelmed’ by NHS licensing of ‘world’s most expensive’ gene therapy

The parents of a 10-month-old baby say they were overwhelmed and cried when they heard that the treatment which could keep him alive had been approved for use by the NHS despite it being labelled the most expensive drug in the world.

Rosie-Mae Walton, 19, and Wes Powell, 22, said “it would mean the absolute world to us” if their son Marley, who has a rare genetic disorder, was eligible for treatment with the US gene therapy Zolgensma after it was licensed for use on the NHS in England.

But they said it is too early to know whether he will be approved for the drug.

Ms Walton and Mr Powell have been raising money for Marley, who has Type 1 spinal muscular atrophy (SMA), to go to the United States for treatment with Zolgensma which has an eye-watering price tag of around £1.8 million for the single dose that is needed.

It's so amazing that it's been approved and can help so many other babies that are diagnosed with SMA

Now the family’s hopes have been raised by the landmark news from the NHS last week.

Ms Walton told the PA news agency: “Our initial reaction when Zolgensma was approved by NHS England was shock and we did cry with being so overwhelmed.

“It’s so amazing that it’s been approved and can help so many other babies that are diagnosed with SMA.”

She said: “We are still unsure if Marley is eligible for Zolgensma and are still waiting on news from our specialists before any decisions can be made.

“We hope that he will be. However, until then we do not know.”

Ms Walton said: “I’ve been in touch with some of Marley’s specialists and they have just confirmed about it being approved. However, it’s just a waiting game for them as much as it is us at this moment in time.”

She said: “It would mean the absolute world to us if Marley was to get Zolgensma treatment.

“He deserves the world and we hope we can give him the treatment to give him the life he deserves and live his life healthier.”

NHS England said last week it had struck a “landmark confidential deal” with US-based manufacturer Novartis Gene Therapies ensuring patients can get the crucial treatment “at a price that is fair to taxpayers”.

Chief executive Sir Simon Stevens said the deal was a “life-changer” children and their families and Health Secretary Matt Hancock called the decision “a game-changer”.

Around 95% of babies with SMA do not live past 18 months without effective treatment.

The condition is similar to motor neurone disease (MND), and makes Marley’s muscles extremely weak, stops him moving his arms and legs, and causes breathing and numerous other life-limiting difficulties.

Studies have shown a one-time intravenous infusion of Zolgensma can help youngster 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, NHS England has said.

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

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.

Ms Walton said: “His muscle movement is improving each day, we often see a little difference. But he still does have his off days and still requires suction every day.

“He has his ventilator on every night and still tolerates wearing that fine.”

In December, PA photographer Joe Giddens, from Leicester, battled hailstorms, freezing rain and 60mph winds to scale England’s highest peak, Scafell Pike, six times in 24 hours in a bid to raise funds for Marley’s trip to the United States.

Ms Walton and Mr Powell, who are from Driffield, East Yorkshire, said: “We are so overwhelmed with every person that has helped raise the amount we have so far and, even though Zolgensma has been approved by NHS England, we would use the money for Marley’s equipment and physio if he is eligible.”

Donations can be made at www.justgiving.com/crowdfunding/marleysmission