Swiss pharmaceutical company Novartis on Friday announced it had received US regulatory approval for a gene therapy that treats a rare childhood disorder and has a price tag of $2.1 million, making it the most expensive drug in history. The company said Zolgensma was a one-time treatment for spinal muscular atrophy, a disease that affects about 1 in 10,000 births and which results in death or the need for permanent ventilation by the age of two in 90 percent of cases. Novartis defended the pricing by saying that gene therapy was a transformative new type of treatment and was 50 percent cheaper than current treatments.
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