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Pricey, But Available

A pricey gene therapy treatment for spinal muscular atrophy (SMA) will be available through the National Health Service in England later this year, the Guardian reports.

SMA, a genetic disorder affecting infants and children, leads to paralysis, breathing problems, and death, Novartis's Zolgensma delivers a working copy of the SMN gene to halt disease progression. The gene therapy was approved in the US in 2019 to treat children under the age of two with certain forms of the disease, as indicated by genetic testing. At the time, the Associated Press noted that the gene therapy was the most expensive medicine at a list price of $2.1 million.

According to the Guardian, the NHS says it has negotiated a discount with the drugmaker, though it is still the most expensive treatment approved by the National Institute for Health and Care Excellence. "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," Simon Stevens, the chief executive of NHS England, tells the Guardian

It adds that about 65 babies in England each year are born with SMA.