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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Related on the SLP
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.
