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The headquarters of the U.S. Food and Drug Administration in Silver Spring, Maryland, Nov. 4, 2009.

2009年11月4日，美国食品药品监督管理局总部位于马里兰州银泉。

Jason Reed | Reuters

杰森·里德 | 路透社

The Food and Drug Administration on Tuesday approved a decades-old prescription vitamin called

食品和药物管理局周二批准了一种已有数十年历史的处方维生素，称为

leucovorin

亚叶酸

as the first treatment for

作为首次治疗方案

a rare genetic disorder

一种罕见的遗传疾病

in certain adults and children.

在某些成人和儿童中。

The move comes months after the Trump administration touted leucovorin as a potential therapy for a broader group of patients with autism spectrum disorder symptoms. The claim sparked skepticism among some in the medical and research community, but fueled excitement among families,

此举出台的几个月前，特朗普政府吹捧了亚叶酸钙可用于更广泛的自闭症谱系障碍症状患者的潜在疗法。这一说法引发了医学界和研究界一些人的质疑，但却让患者家属感到兴奋不已，

spiking prescriptions

尖峰处方

of the drug in the U.S.

美国的药品

One FDA official told reporters on Monday that 'we don't have sufficient data to say that we could establish efficacy for autism more broadly,' but said the agency is open to interest from companies in studying leucovorin in the autism population.

一位 FDA 官员周一告诉记者：“我们没有足够的数据来说明我们可以更广泛地确定对自闭症的有效性，”但表示该机构有兴趣开放公司研究叶酸在自闭症群体中的应用。

The medication, also referred to as folinic acid, is a synthetic form of vitamin B9 that has been used to treat the toxic side effects of chemotherapy. Just a handful of small trials have suggested that leucovorin could be effective as an off-label treatment for children with autism, and some families have reported that it helped their nonverbal kids develop more language and social skills..

这种药物也被称为亚叶酸，是一种合成形式的维生素B9，已被用于治疗化疗的毒副作用。只有少数小型试验表明，亚叶酸作为一种标签外治疗对自闭症儿童可能有效，一些家庭报告称，它帮助他们的非语言孩子发展出更多的语言和社交技能。

FDA officials, who requested anonymity to discuss the decision, told reporters on Monday that they started with a broad review of leucovorin as an autism treatment before narrowing its approval to a smaller population with cerebral folate deficiency, a rare genetic mutation that prevents folate – a key vitamin – from properly reaching the brain. .

FDA 官员周一告诉记者，他们要求匿名以便讨论这一决定，他们最初对亚叶酸作为自闭症治疗方法进行了广泛的审查，之后将其批准范围缩小到脑叶酸缺乏症患者，这是一种罕见的基因突变，会阻止关键维生素叶酸正常到达大脑。

The condition shares overlapping features with autism, typically develops in young children under age 2 and can cause severe developmental delays, seizures, a lack of muscle control and other serious neurological complications.

这种情况与自闭症有共同的特征，通常在两岁以下的幼儿中发展，并可能导致严重的发育迟缓、癫痫发作、缺乏肌肉控制和其他严重的神经系统并发症。

The officials said the FDA found that using leucovorin in patients with that condition produced the 'highest quality data' to support an expanded approval, which will apply to both generic versions of the drug and GSK's old branded medication,

官员们表示，FDA 发现，在患有该疾病的患者中使用亚叶酸产生了支持扩大批准的“最高质量数据”，这将适用于该药物的仿制药版本和 GSK 的老牌药物。

Wellcovorin

Wellcovorin

.

。

'That was the data where we saw the largest effect sizes,' one FDA official said on the call. 'So we narrowed in on that population, just because we felt like that was the strongest, both scientific rationale and also the largest treatment effects that could be used to then overcome some of the limitations in the data sources.'.

“这是我们在数据中看到最大效应值的地方，”一位FDA官员在电话中说。“因此，我们聚焦于该人群，因为我们认为这是最有力的，无论是科学依据还是可以用来克服数据来源中一些限制的最大治疗效果。”

The approval was based on a systematic review of published literature on the area, including patient case reports, but not a randomized controlled clinical trial. The same official acknowledged there can be biases with systematic reviews, but emphasized that the treatment effects were so large that they outweighed those concerns. .

该批准是基于对已发表的该领域文献的系统性回顾，包括患者病例报告，但并非随机对照临床试验。同一位官员承认系统性回顾可能存在偏倚，但强调治疗效果非常显著，足以抵消这些担忧。

The FDA is encouraging existing manufacturers of leucovorin to increase production to match higher demand for the drug, the officials added. While GSK originally marketed the drug from 1983 until 1997, the company said in September that it

官员们补充说，FDA正在鼓励现有的亚叶酸制造商提高产量以满足该药物更高的需求。虽然葛兰素史克公司最初在1983年至1997年期间销售该药物，但该公司在九月份表示

has no plans to re-launch and manufacture

没有重新启动和制造的计划

the product itself.

产品本身。

In a release on Tuesday, Dr. Tracy Beth Hoeg, acting director of the FDA's Center for Drug Evaluation and Research, said the approval demonstrates the FDA's commitment to 'rapidly identifying effective treatments for ultra rare diseases while maintaining the same evidentiary standards for approval.'.

在周二的发布会上，FDA药物评估和研究中心代理主任特雷西·贝丝·霍格博士表示，此次批准体现了FDA“在保持相同审批证据标准的同时，快速识别超罕见疾病有效治疗方法”的承诺。

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将CNBC设置为Google上的首选来源，不错过商业新闻最值得信赖的名字的任何时刻。