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Zolgensma wins FDA green light in child SMA

May 28, 2019 / Anna Smith

The U.S. Food and Drug Administration (FDA) has approved Novartis’ Zolgensma (onasemnogene abeparvovec-xioi) at a cost of $2.1 milllion per treatment. The drug is the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), which is the most severe form of SMA and a leading genetic cause of infant mortality.The price tag has garnered substantial criticism, as even the split payment structure adds up to $425,000 per year over five years. Novartis has announced that it will give ‘partial rebates’ if the treatment doesn't work.The approval marks a “milestone in the transformational power of gene and cell therapies to treat a wide range of diseases,” said acting FDA commissioner Ned Sharpless. He continued, “With each new approval, we see this exciting area of science continue to move beyond the concept phase into reality. The potential for gene therapy products to change the lives of those patients...