The most expensive drug in the U.S. is now Lenmeldy, a $4.25 million gene therapy the FDA approved March 18 for children with a rare genetic disease. Metachromatic leukodystrophy manifests into the loss of motor and cognitive function, and early death, according to the FDA. The first and only one-time medicine for patients is Lenmeldy (atidarsagene autotemcel).

美国目前最昂贵的药物是Lenmeldy，这是一种3月18日FDA批准用于罕见遗传病儿童的425万美元基因疗法。据FDA称，变色性脑白质营养不良表现为运动和认知功能丧失以及早期死亡。Lenmeldy（atidarsagene autotemcel）是第一种也是唯一一种用于患者的一次性药物。

Its manufacturer, Orchard Therapeutics, said the drug's wholesale acquisition cost is $4.25 million. In a March 20 news release, Orchard said the price tag 'is reflective of the value the therapy may deliver to eligible patients and their families, as well the potential long-term impact [the] treatment may have on overall healthcare utilization, minimization of productivity loss for caregivers and life opportunities for patients.'In a trial, 37 children received Lenmeldy and experienced a significant reduction in the risk of severe motor impairment and death compared to untreated children.

其制造商Orchard Therapeutics表示，该药物的批发收购成本为425万美元。Orchard在3月20日的新闻稿中表示，价格标签“反映了该疗法可能为符合条件的患者及其家人带来的价值，以及该疗法可能对整体医疗保健利用率产生的潜在长期影响，最大程度地减少护理人员的生产力损失和患者的生活机会。”在一项试验中，37名儿童接受了Lenmeldy治疗，与未经治疗的儿童相比，严重运动障碍和死亡的风险显着降低。

At 5 years old, 71% of treated children could walk without assistance. All study participants who had pre-symptomatic late infantile MLD were alive at 6 years old, compared to 58% of children in the control group. Before its approval, the Institute for Clinical and Economic Review said the drug would be cost effective if priced between $2.3 million and $3.9 million. Other chart-topping medications include Hemgenix, a $3.5 million hemophilia B therapy; Elevidys, a $3.2 million muscular dystrophy drug; and Skysona, a $3 million medicine for adrenoleukodystrophy, according to CNN. .

在5岁时，71%的接受治疗的儿童可以在没有帮助的情况下行走。所有有症状的晚期婴儿MLD的研究参与者在6岁时都还活着，而对照组中有58%的儿童还活着。在批准之前，临床与经济评论研究所表示，如果该药物的价格在230万美元至390万美元之间，将具有成本效益。其他排名靠前的药物包括Hemgenix，一种350万美元的血友病B疗法；Elevidys，一种320万美元的肌肉萎缩症药物；据美国有线电视新闻网报道，Skysona是一种价值300万美元的肾上腺脑白质营养不良药物。。