Orchard $4.3M gene therapy becomes most expensive in the world

2024-03-20

基因疗法上市批准并购

Orchard Therapeutics, now a unit of Kyowa Kirin of Japan, has set the price for its newly approved gene therapy for metachromatic leukodystrophy (MLD) at $4.25 million, claiming the title of the most expensive drug in the world.

Lenmeldy snagged FDA approval this Monday for children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile MLD — all of which previously had no treatment options beyond supportive and end-of-life care. The disease presents a grim prognosis, with about 50% of affected individuals not surviving more than five years after diagnosis.

According to Orchard, the pricing mirrors the anticipated benefits the one-time therapy could provide to patients and their families, along with its potential to reduce long-term health care costs. The wholesale acquisition cost is not the definitive cost for patients, as final expenses are influenced by insurance coverage.

Orchard says it now engaging with both commercial and governmental insurance providers to establish agreements based on the therapy's outcomes and value, aiming to secure coverage.

In assessing the therapy's price, the Institute for Clinical and Economic Review, a third-party evaluator of drug pricing, endorsed a maximum health-benefit value of up to $3.94 million for the treatment. ICER conducts independent evaluations of drug values to influence health care policies and pricing. Their valuations indicate if a therapy's price aligns with its benefits, considering health outcomes and economic impacts. These assessments guide negotiations between insurers and manufacturers and inform drug pricing discussions.

Before Lenmeldy, the most expensive drug was CSL Behring's hemophilia B gene therapy, Hemgenix, priced at $3.5 million following its approval in November 2022.

更多内容，请访问原始网站

文中所述内容并不反映新药情报库及其所属公司任何意见及观点，如有版权侵扰或错误之处，请及时联系我们，我们会在24小时内配合处理。

机构