The FDA has approved Zolgensma, a gene replacement therapy drug from pharmaceutical company Novartis that treats spinal muscular atrophy for use in children younger than 2 years-old.
Why this matters: With a price tag of $2.1 million USD, it becomes the world’s most expensive drug. The drug attacks a debilitating genetic disease that has resulted in infant deaths.
Know more: Novartis said it will allow health insurance companies to pay for Zolgensma’s $2.1 million price, which does not factor in potential rebates or discounts, over 5 years.
Behind the numbers: It is worth noting that Novartis acquired AveXis, the biotech firm that developed the gene therapy drug, for $8.7 billion USD last year, and investors are looking for a return on their investment.