The Most Expensive Drug From Novartis Associated With Two Deaths: Report

The Most Expensive Drug From Novartis Associated With Two Deaths: Report
  • Two children with spinal muscular atrophy have died after receiving Novartis AG's NVS Zolgensma, a one-time gene therapy, spotlighting its risks and questioning the safety of genetic medicines like it.
  • The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan.
  • According to GoodRx Holdings Inc's GDRX report, Zolgensma is the most expensive drug in America, with an average one-time cost of $2.1 million.
  • The patients developed acute liver failure five to six weeks after one-time gene therapy infusion and one to ten days after corticosteroid tapering, which is done to ensure proper liver function.
  • According to STAT, Novartis began notifying physicians and payers this week, which first reported the news.
  • "While this is important safety information, it is not a new safety signal, and we firmly believe in the overall favorable risk/benefit profile of Zolgensma," Novartis said
  • Zolgensma was approved in the U.S. in May 2019, becoming just the second gene therapy for an inherited disease cleared by the FDA. 
  • The risk of liver injury, in particular, is mentioned on the FDA's product labeling, which instructs doctors to assess liver function before infusing Zolgensma and to administer steroids before and after to manage increases in liver enzyme counts.
  • Two previous cases of acute liver failure have been reported following Zolgensma treatment.
  • Price Action: NVS shares are down 0.78% at $84.91 during the market session on the last check Friday.

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