一個療程1400萬元！史上最昂貴的藥物在美通過審批！

US regulators approve world"s most expensive drug - costing ￡1.6m

美國監管機構批准了世界上最昂貴的藥物——花費160萬英鎊

來源：天空新聞 翻譯：世界播

瑞士諾華公司新葯的價格引發了批評

Swiss pharmaceutical giant Novartis has won approval for a new gene therapy drug which is priced at a record ￡1.67m per treatment.

瑞士製藥巨頭諾華的一種新型基因治療藥物獲得批准，每次治療的價格達到創紀錄的167萬英鎊。

Named Zolgensma, the drug has been approved by US regulators for treating babies with spinal muscular atrophy (SMA) - a rare and often fatal disease.

該藥物名為Zolgensma，已被美國監管機構批准用於治療患有脊髓性肌萎縮症的嬰兒。脊髓性肌萎縮症是一種罕見且往往致命的疾病。

The new one-time treatment uses a virus to replace the defective gene which causes SMA with a copy that functions properly.

這種新的一次性治療方法使用一種病毒，用一種功能正常的複製體替換導致脊髓性肌萎縮症的缺陷基因。

The regulatory approval allows Zolgensma to be used on babies with the deadliest form of the disease as well as ones whose lives could be impacted later.

監管部門的批准允許Zolgensma被用於患有該病最致命形式的嬰兒以及那些日後可能受到影響的嬰兒。

Babies affected by the most severe form of the progressive condition rarely survive beyond two years of age.

嬰兒如果患有這種進行性疾病的最嚴重形式，很少能活過兩歲。

"This is potentially a new standard of care for babies with the most serious form of SMA," pediatric neurologist Dr Emmanuelle Tiongson said.

兒科神經學家伊曼紐爾·廷格森博士說：「對於患有最嚴重脊髓性肌萎縮症的嬰兒來說，這可能是一種新的標準治療。」

"The job now is trying to negotiate with insurers that this would be a long-term savings," added Dr Tiongson, who provided Zolgensma to patients under an expanded access program.

廷格森博士補充說：「現在要做的就是努力與保險公司協商，這將是長期的成本節約。」廷格森博士根據擴充療程方案向患者提供Zolgensma。

Executives at the pharmaceutical giant defended their price of the drug and claimed that the one-time treatment, which is delivered via spinal infusion, is more valuable than alternatives which cost hundreds of thousands of dollars a year.

這家製藥巨頭的高管們為這種藥物的價格進行了辯護，稱這種通過脊髓注射進行的一次性治療比每年花費數十萬美元的替代療法更有價值。

The cost of gene therapy drugs has been a subject of hot debate. An independent review in April by the Institute for Clinical and Economic Review (ICER) in the US claimed that Novartis was overestimating the value of Zolgensma.

基因治療藥物的成本一直是一個熱門話題。今年4月，美國臨床與經濟審查協會發表的一篇獨立評論稱，諾華高估了Zolgensma的價值。

But following the approval on Friday, the ICER retracted this claim, saying that based on additional clinical data provided by Novartis it now believed that Zolgensma was, while still expensive, within range for being cost-effective.

但在周五獲得批准後，臨床與經濟審查協會收回了這一說法，稱基於諾華提供的額外臨床數據，它現在認為，Zolgensma雖然還是價格昂貴，但在成本效益範圍之內。

Novartis has also submitted the drug for review to the European Medicines Agency and a decision from the regulator is expected in the coming months.

諾華還將此葯提交歐洲藥物品管理局進行審查，預計該監管機構將在未來幾個月做出決定。

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