Novartis gets US approval for $2.1 million gene therapy

  • Bizline

May 26, 2019-

WASHINGTON: 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. But the announcement comes as the administration of US President Donald Trump has vowed to tackle soaring drug costs. Novartis defended the pricing by saying that gene therapy was a transformative new type of treatment and was 50 percent cheaper than current treatments. “Zolgensma is a historic advance for the treatment of SMA and a landmark one-time gene therapy,” Novartis CEO Vas Narasimhan said in a statement, which added that the company was working with government and insurers to accelerate coverage. The total cost will be $2.125 million payable over five years at $425,000 per year. Zolgensma works by providing a functional copy of the defective gene responsible for SMA to halt the disease’s progression via a one-time intravenous infusion. (AFP)

Published: 26-05-2019 09:23

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