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

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

诺华最昂贵的药物与两人死亡有关：报道
Benzinga Real-time News ·  2022/08/12 11:16
  • Two children with spinal muscular atrophy have died after receiving Novartis AG's (NYSE: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 (NASDAQ: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.
  • 两名脊髓性肌萎缩症儿童在接受治疗后死亡诺华制药(纽约证券交易所股票代码：NVS)Zolgensma是一种一次性基因疗法，它强调了其风险，并质疑类似它的基因药物的安全性。
  • 这些由急性肝功能衰竭导致的死亡发生在俄罗斯和哈萨克斯坦。
  • 根据GoodRx Holdings Inc.的纳斯达克：GDRX报道，佐根斯马是美国最昂贵的药物，平均一次性成本为210万美元。
  • 患者在一次性基因治疗输注后5至6周和皮质类固醇缩减后1至10天出现急性肝功能衰竭，这是为了确保正常的肝功能。
  • 根据STAT的数据，诺华公司本周开始通知医生和付款人，他们最先报道了这一消息。
  • 诺华公司表示：“虽然这是重要的安全信息，但并不是一个新的安全信号，我们坚信Zolgensma的总体风险/收益状况良好。”
  • Zolgensma于2019年5月在美国获得批准，成为FDA批准的第二种遗传病基因疗法。
  • FDA的产品标签上特别提到了肝脏损伤的风险，该标签指示医生在注射Zolgensma之前评估肝功能，并在注射Zolgensma前后使用类固醇以控制肝酶计数的增加。
  • 据报道，在佐根斯马治疗后，之前有两例急性肝功能衰竭。
  • 价格行动：在周五的最后一个交易日，NVS的股价下跌了0.78%，至84.91美元。
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