Zolgensma, a drug made by the pharmaceutical company Novartis, that offers the most tantalizing hope [for children born with spinal muscular atrophy (SMA).]
Zolgensma is administered via a single, hour-long intravenous infusion that has the potential to stop the degeneration caused by SMA in its tracks.
If given at birth, before symptoms develop, doctors believe that the one-shot treatment could approach a cure. But the older the baby, the less effective and riskier the treatment, leaving families in a desperate race against time. And there’s another hurdle: it costs $2.1m (£1.6m) a dose. When it was licensed in the US in 2019, this made it the most expensive drug in the world.
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To address global access, since 2020 Novartis has committed to offer free treatment to 100 babies each year in countries where Zolgensma has not received regulatory approval, via a lottery-style draw – one where the stakes are unbearably high.
The initiative, in which babies are selected randomly by a third party, has had a mixed reaction. The campaigning group Just Treatment likened the draw to a “real life Hunger Games”, while the charity SMA Europe expressed alarm at the prospect of babies competing for a lifesaving treatment.
