Life-saving gene therapy to treat babies with rare diseases made available in the NHS

With the world’s most expensive drug approved for use in the UK NHS, up to 80 babies born with rare muscle-depleting disorders can receive life-changing treatment each year.

Babies born with spinal muscular atrophy (SMA) are a deadly condition that often affects the spinal nerves and causes paralysis, giving them access to the one-time gene therapy Zolgensma. ..

The drug, which replicates the missing gene and restores nerve and muscle function, is reported to have a list price of approximately £ 1.8 million per dose.

However, the NHS England said it has entered into a “breakthrough confidential transaction” that allows patients to receive critical treatment “at a fair price for taxpayers.”

Sir Simon Stevens, Chief Executive Officer of NHS England, said:

“Spinal muscular atrophy is the leading cause of genetic death in infants, so NHS England can take advantage of this treatment while negotiating hard behind the scenes to ensure fair prices for taxpayers. I moved the mountain like. “

Life expectancy for babies born with severe type 1 SMA (the most common condition) is only two years.

However, studies have shown that a single intravenous infusion of Zorgensma helps the baby breathe without a ventilator, get up without help, and crawl.

NHS England said as many as 80 babies and toddlers could benefit from life-changing treatments each year.

The drug contains a replica of the lost gene SMN1 and the active ingredient onasemnogene aveparbovec enters the nerves and restores the gene. This produces the proteins needed to control nerve function and muscle movement.

Health Minister Matt Hancock said: “This drug is a breakthrough for babies born with this rare muscle-depleting disease. We are very pleased that the NHS will soon be able to offer this treatment to babies and toddlers.

“Every child is worth enjoying a painless, happy and healthy future. The NHS is working tirelessly to make this happen.”

The deal was secured with Novartis Gene Therapy, a US-based manufacturer.

The National Institute for Health and Care Excellence (Nice) has published a draft guidance recommending treatment with Zolgensma for infants up to 12 months of age with type 1 SMA.

Nice’s statement said: “Despite the high cost of treatment, infants have extraordinary benefits and may reach milestones in normal infancy development, so their use in the NHS is recommended.

“The limited study data for children aged 7 to 12 months requires that treatment be considered by interdisciplinary clinical teams across the country.”

Sir Simon said: Those who are now capable of real medical progress. “

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