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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

諾華最昂貴的藥物與兩人死亡有關：報道
Benzinga Real-time News ·  2022/08/12 23:15
  • 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.
  • 兩名脊髓性肌萎縮症兒童在接受治療後死亡諾華製藥(紐約證券交易所股票代碼：NVS)Zolgensma是一種一次性基因療法，它強調了其風險，並質疑類似它的基因藥物的安全性。
  • 這些由急性肝功能衰竭導致的死亡發生在俄羅斯和哈薩克斯坦。
  • 根據GoodRx Holdings Inc.的納斯達克：GDRX報道，佐根斯馬是美國最昂貴的藥物，平均一次性成本為210萬美元。
  • 患者在一次性基因治療輸注後5至6周和皮質類固醇縮減後1至10天出現急性肝功能衰竭，這是為了確保正常的肝功能。
  • 根據STAT的數據，諾華公司本週開始通知醫生和付款人，他們最先報道了這一消息。
  • 諾華公司表示：“雖然這是重要的安全信息，但並不是一個新的安全信號，我們堅信Zolgensma的總體風險/收益狀況良好。”
  • Zolgensma於2019年5月在美國獲得批准，成為FDA批准的第二種遺傳病基因療法。
  • FDA的產品標籤上特別提到了肝臟損傷的風險，該標籤指示醫生在注射Zolgensma之前評估肝功能，並在注射Zolgensma前後使用類固醇以控制肝酶計數的增加。
  • 據報道，在佐根斯馬治療後，之前有兩例急性肝功能衰竭。
  • 價格行動：在週五的最後一個交易日，NVS的股價下跌了0.78%，至84.91美元。

譯文內容由第三人軟體翻譯。


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