NHS England: Most expensive drug in the world is now accessible

A drug that treats babies and young children with a rare and frequently life-limiting degenerative disorder will be available this year on the NHS England.

Zolgensma is the most expensive in the world, costing £1.79 million per dose, and replaces the function of the faulty SMN1 gene, which prevents the development of Spinal Muscular Atrophy (SMA).

SMA causes a loss of movement, muscle weakness and paralysis in some young children. It is the leading genetic cause of infant deaths and affects one in every 10,000 live births.

Around 80 infants per year will receive this drug through a single dose. The active ingredient, onasemnogene abeparvovec, restores the gene and produces proteins essential for muscle movement and nerve function.

Zolgensma does not cure the SMA disorder, but it does hinder the progression of SMA type one, the most severe form, which leaves babies with a life expectancy of two years.

Through the use of this drug, these infants can breathe without a ventilator, sit up, crawl and even walk. These benefits last longer than five years after the single treatment.

Chief Executive of NHS England Sir Simon Stevens said, “SMA is the leading genetic cause of death among babies and young children, which is why NHS England has moved mountains to make this treatment available, while successfully negotiating hard behind the scenes to ensure a price that is fair to taxpayers.”

The price England bought the drug for it confidential. However, according to the NHS, a hefty discount has been negotiated.

Currently, the NHS is working out which specialist services will be able to and should administer the drug.

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