Five-month-old baby receives gene therapy Zolgensma on NHS

pharmafile | June 1, 2021 | News story | Manufacturing and Production, Sales and Marketing  

A five-month-old baby is one of the first patients to receive Zolgensma, the world’s most expensive drug, on the NHS.

Zolgensma is a one-off gene therapy for babies with spinal muscular atrophy (SMA).

If left untreated, children born with SMA would otherwise have a life expectancy of less than two years.

Around 40 children are born with the most severe form of SMA every year.

The treatment, made by Novartis, is also the most expensive drug in the world, but the NHS has negotiated a confidential discount which means dozens of infants can be treated each year with Zolgensma.

Five-month-old baby Arthur is one of these patients. He finds it difficult to move his arms and legs and cannot lift his head, so some permanent damage has already been done.

The hope is the one-off treatment will stabilise his condition and prevent further deterioration.

Evelina Children’s Hospital in London, where Arthur is being treated, is one of a handful of centres which will be offering Zolgensma.

Four specialist NHS centres have now been commissioned across the country. 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 the Evelina told the BBC: “20 years ago a baby born with SMA would have a very shortened life expectancy.

“Now, we have the potential to enable children to acquire motor skills such as rolling and sitting, and even standing-walking, that would have been impossible without treatment for SMA.”

NHS England Chief Executive Sir Simon Stevens said it is “fantastic news that this revolutionary treatment is now available for babies and children like Arthur on the NHS”.

Sally-Anne Tsangarides, General Manager at Novartis Gene Therapies in the UK, said it is a “hugely important step for babies in England”.

She continued, “We thank all those who have been involved in the landmark agreement that has made it possible.”

Despite the extremely high list price, both NICE and the Scottish Medicines Consortium recommended the NHS fund the treatment.

Trials have shown it produces lasting benefits, which endure at least five years, and perhaps will prove to be permanent.

The only other treatment for SMA involves costly spinal infusions which need to be given at least three times a year.

Zolgensma contains a healthy copy of a missing or faulty gene called SMN1. This is then inserted to a harmless virus which delivers the replacement gene into the nucleus of motor neuron cells.

This prevents the cells from gradually dying, and the now healthy motor neuron cells can start producing the missing SMN protein which is vital for muscle function.

Arthur’s father, Reece Morgan said: “Our hope is that he can have the best possible life in terms of his movement.

“We don’t know, but we’re just going to try as best as we can to give him everything he possibly needs.”

Lilly Subbotin

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