世界上最昂贵的药物是基因治疗,价值200万美元
World’s Costliest Medicine Is Gene Treatment Priced at $2 Million

世界上最昂贵的药物是基因治疗,价值200万美元

World’s Costliest Medicine Is Gene Treatment Priced at $2 Million

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世界上最昂贵的药物是基因治疗,价值200万美元

World’s Costliest Medicine Is Gene Treatment Priced at $2 Million

2019-06-06

美国官员批准了史上最昂贵的药物——一种治疗罕见儿童疾病的基因疗法,价值210万美元。


美国官员批准了史上最昂贵的药物——一种治疗罕见儿童疾病的基因疗法,价值210万美元。
U.S. officials have approved the costliest medicine in history – a $2.1-million gene therapy that treats a rare childhood disease.

基因治疗是一种将健康的遗传物质导入细胞以取代异常基因的医学治疗方法。
Gene therapy is a medical treatment in which healthy genetic material is introduced into cells to replace abnormal genes.

这项新基因疗法针对的是一种名为脊髓性肌萎缩症(SMA)的疾病,这种疾病会严重削弱儿童的肌肉。
The new gene therapy targets a disease called spinal muscular atrophy or SMA, which severely weakens a child’s muscles.

这种名为佐尔根斯玛(Zolgensma)的药物是由瑞士制药公司诺华研发。
The treatment, called Zolgensma, was developed by Swiss drugmaker Novartis.

随着时间的推移,脊髓性肌萎缩症会使儿童无法移动、吞咽或呼吸。
Over time, SMA can make it impossible for the child to move, swallow or breathe.

每1万名新生儿中就有一人感染这种疾病。
The disease affects about 1 in 10,000 births.

在90%的病例中,这种情况会导致死亡,或者在两岁前需要永久性的呼吸辅助。
In 90 percent of cases, the condition causes death or the need for permanent breathing assistance by age two.

脊髓性肌萎缩症是婴儿遗传死亡的主要原因。
SMA is the leading cause of genetic infant death.

美国食品和药物管理局最近批准可以用佐尔根斯玛用于治疗两岁以下患有脊髓性肌萎缩症的儿童。
The U.S. Food and Drug Administration or FDA recently approved Zolgensma for use in children who are under the age of two with SMA.

这项批准覆盖的患者包括患有这种最致命疾病,以及那些随着时间推移发育较慢的婴儿。
The approval covers babies with the deadliest kind of the disease as well as those with forms that develop more slowly over time.

诺华表示,佐尔根斯玛旨在通过替换患者异常或缺失的基因来阻止疾病的进展,从而修复脊髓性肌萎缩症的遗传原因。
Novartis says Zolgensma is designed to repair the genetic cause of SMA by replacing a patient’s abnormal or missing gene to halt progression of the disease.

一次性治疗约1小时即可完成。
The one-time treatment can be performed in about an hour.

诺华为基因治疗的高成本进行了辩护。
Novartis has defended the high cost of the gene therapy.

它说,这种疗法代表了一种新型的“转化医学”,为未来的基因疗法打开了大门。
It says the treatment represents a new kind of "transformational medicine" that opens the door to future gene therapies.

该公司还表示,210万美元的治疗费用比其他持续数年的脊髓性肌萎缩症治疗费用低50%左右。
It also said the $2.1-million therapy was about 50 percent less than other SMA treatments that can be ongoing for years.

诺华表示,它将让医疗保险公司在未来5年内分摊42.5万美元的保费。
Novartis said it will let health insurance companies break up the cost in payments of $425,000 over five years.

如果治疗无效,该公司还计划退款。
It also plans to give money back if the treatment does not work.

美国食品和药物管理局表示,该药物的安全性已经在36名年龄在两周至八个月之间的患者身上进行了测试。
The FDA said the drug’s safety had been tested in completed and ongoing trials involving 36 patients between the ages of two weeks and eight months.

治疗效果的大多数证据都是基于正在进行的试验。
Most evidence of the treatment’s effectiveness is based on the ongoing trials.

在病人测试中,患有最严重的脊髓性肌萎缩症婴儿在出生后6个月内获得了佐尔根斯玛的治疗,他们现在的肌肉问题不多。
In patient testing, babies with the most severe form of SMA who got Zolgensma within 6 months of birth had limited muscle problems.

那些在生命早期接受治疗的人表现最好。
Those who got the treatment very early in life did the best.

服用佐尔根斯玛六个月后的婴儿肌肉不再失去控制,但药物无法治疗已经造成的损伤。
Babies who were given Zolgensma after six months stopped losing muscle control, but the medicine was not able to correct damage already done.

试验发现,这种疗法“显示出他们达到发育性运动里程碑的能力有显著改善。”
The trial found the treatment "demonstrated significant improvement in their ability to reach developmental motor milestones."

包括控制头部和在没有支撑的情况下坐着的能力。
These included head control and the ability to sit without support.

美国批准的另一种治疗脊髓性肌萎缩症的药物叫做斯宾拉沙(Spinraza)。
The one other medicine for SMA approved in the U.S. is a drug called Spinraza.

它不能一次性治愈,必须每四个月治疗一次。
Instead of a one-time treatment, it must be given every four months.

斯宾拉沙的制造商、总部位于美国的生物基因公司表示,第一年的治疗费用为75万美元,之后每年收费35万美元。
The U.S.-based Biogen, Spinraza’s maker, charges $750,000 for the first year of treatment, followed by $350,000 per year after that.

独立的非营利组织临床与经济评论协会评估了昂贵新药的价值。
The independent not-for-profit group Institute for Clinical and Economic Review rates the value of costly new medicines.

该组织表示,他们认为佐尔根斯玛的价格是可以接受的,因为它“极大地改变了受这种毁灭性疾病影响的家庭的生活”。
The group said it considers the price of Zolgensma acceptable because it greatly "transforms the lives of families affected by this devastating disease."

虽然现在还不知道这种疗法的效果能持续多久,但一些医生希望这种疗法能持续一辈子。
While it is too early to know how long the effectiveness of the treatment will last, some doctors have hope that it could last a lifetime.

杰里·门德尔博士是俄亥俄州哥伦布市全国儿童医院的神经学家。
Dr. Jerry Mendell is a neurologist at Nationwide Children’s Hospital in Columbus, Ohio.

他领导了一项关于佐尔根斯玛治疗的早期病人研究。
He led one of the early patient studies involving Zolgensma.

当被问及他是否相信这种药物会继续产生长期效果时,门德尔告诉美联社,“情况正朝着这个方向发展。”
When asked whether he believes the drug will continue to have long-lasting effects, Mendell told the Associated Press, "it’s beginning to look that way."

他指出,一些接受婴儿治疗的儿童现在已经四、五岁了,没有任何患病迹象。
He noted that some of those treated as babies are now 4 or 5 years old with no signs of the disease.

布莱恩·林恩为您播报。
I’m Bryan Lynn.