Kate Cardente holds her then 3-month-old daughter Ainsley as she undergoes gene therapy for Spinal Muscular Atrophy (SMA). SMA is a disease that is the primary genetic cause of death in babies. Ainsley receives a one-time infusion from Zolgensma.

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LONDON – A drug that has been classified as “the most expensive drug in the world” has been approved by the UK’s National Health Service. This could be life changing for babies and children with a rare genetic disorder.

The innovative gene therapy called “Zolgensma” is not cheap at a list price of £ 1.79 million (US $ 2.48 million) per dose, NHS England said in a statement on Monday.

The drug is used in babies and young children who suffer from spinal muscular atrophy, a rare and often fatal genetic condition that causes paralysis, muscle weakness, and progressive loss of movement.

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

The drug will be available through the UK healthcare system, which will provide free medical care at the time of delivery, “at a price that will suit taxpayers under a landmark confidential agreement from NHS England,” CEO Simon Stevens announced Monday. NHS England did not disclose the price paid.

The NHS is primarily funded by general government taxes, so the drugs and treatments it approves and use must undergo rigorous analysis to verify their cost-effectiveness.

Studies have shown that Zolgensma, made by Novartis Gene Therapies (part of the US drug Novartis), helps babies reach milestones such as breathing without a ventilator, sitting alone, and crawling and walking after a single one Infusion to achieve treatment.

The latest data suggest that Zolgensma can provide young children with type 1 SMA with rapid and sustained improvement in motor function and extend their lives.

Up to 80 babies and toddlers a year could potentially benefit from gene therapy, according to the NHS.

Life changer

Stevens said the deal was “a life changer for teenagers with this cruel disease and for their families”.

“Spinal muscular atrophy is the leading genetic cause of death in babies and young children. That’s why the NHS England has moved mountains to provide this treatment while successfully negotiating behind the scenes to ensure a price that would be fair to taxpayers.”

He said the deal shows that while health services remain under pressure from the Covid-19 pandemic, the NHS still “cares for millions of other patients for whom real medical advances are now possible”.

The agreement with Novartis Gene Therapies secures the drug for NHS patients in England at a significant confidential discount and paves the way for the National Institute for Excellence in Health and Care (NICE) – the public body that issues guidelines on drug cost-effectiveness and Treatments used by the NHS – to post draft guidance recommending treatment with Zolgensma.

The terms of the contract mean that some young children who currently do not meet the NICE referral criteria may also be considered for treatment by a national clinical team made up of the country’s leading experts in the treatment of SMA.

The NHS said it was ready to accelerate the rollout of the highly complex and innovative gene therapy and would not wait for NICE to publish final guidelines to get started. This approach is supported by NICE as it is important to have the one-time treatment as early as possible, he added.

The drug’s approval is the second medical treatment now available for adolescents with SMA. It added that future treatment options also look promising, as another SMA drug is currently under review by NICE.