What’s the world’s most expensive drug?

One-off treatment for spinal muscular atrophy in young children gains FDA approval

Gene therapy drug Zolgensma has become the world's most expensive drug, with the one-time treatment priced at $US2,125,500 ($A3m).

ill child

Swiss drugmaker Novartis has won US approval for its gene therapy Zolgensma for spinal muscular atrophy, the leading genetic cause of death in infants.

Novartis executives defended the price, saying that a one-time treatment was more valuable than expensive long-term treatments that cost several hundred thousand dollars a year.

The US Food and Drug Administration has approved Zolgensma for children under the

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