Amylyx Pharmaceuticals 在临床试验失败后撤回 ALS 药物

Amylyx Pharmaceuticals 宣布裁员 70%，并在临床试验显示其 ALS 药物 Relyvrio/Albrioza 无效后自愿撤回该药物。此举是在美国食品和药物管理局基于一项试验对该药物进行有争议的批准之后发生的。该公司已与监管机构启动移除程序，并将在过渡期间向现有患者提供免费药物获取。这一决定很不寻常，因为药物从市场上撤下通常是不常见的。

Amylyx Pharmaceuticals Withdraws ALS Drug from Market

Amylyx Pharmaceuticals 从市场上撤回 ALS 药物

In a significant development, Amylyx Pharmaceuticals has announced its decision to voluntarily remove its drug, Relyvrio (Albrioza in Canada), from the market. This move comes less than a month after the release of a clinical trial demonstrating the inefficacy of the once-promising treatment for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease.

Amylyx Pharmaceuticals 宣布决定自愿将其药物 Relyvrio（加拿大 Albrioza）撤出市场，这是一项重大进展。这一举措是在一项临床试验发布后不到一个月做出的，该试验表明曾经有希望的肌萎缩侧索硬化症（ALS）（也称为卢伽雷氏病）治疗方法无效。

The Cambridge, Massachusetts-based company initiated the process of withdrawing the drug with regulators on Thursday. Effective immediately, Relyvrio will no longer be available for new patients. However, existing patients will have the option to transition to a "free drug program." Amylyx will also implement a 70% reduction in its workforce.

这家总部位于马萨诸塞州剑桥的公司周四与监管机构启动了撤回该药物的程序。立即生效，Relyvrio 将不再对新患者开放。然而，现有患者可以选择过渡到“免费药物计划”。 Amylyx 还将裁员 70%。

"While this is a challenging time for the ALS community, we reached this path forward in collaboration with affected stakeholders and in keeping with our unwavering commitment to individuals living with ALS and other neurodegenerative diseases," stated Joshua Cohen and Justin Klee, co-chief executive officers of Amylyx, in a joint statement.

联席首席执行官约书亚·科恩 (Joshua Cohen) 和贾斯汀·克利 (Justin Klee) 表示：“虽然对于 ALS 社区来说这是一个充满挑战的时期，但我们与受影响的利益相关者合作，并遵循我们对 ALS 和其他神经退行性疾病患者坚定不移的承诺，共同迈出了这条道路。” Amylyx 的执行官在一份联合声明中表示。

The U.S. Food and Drug Administration's (FDA) approval of Relyvrio in 2022 sparked controversy due to its reliance on a single clinical trial. FDA staff and external experts raised concerns about the drug's effectiveness. A larger trial, the results of which were unveiled last month, revealed that the benefit to patients was not even as reliable as a coin toss. The company's stock value plummeted by 80% as a result.

美国食品和药物管理局 (FDA) 于 2022 年批准 Relyvrio，因其依赖单一临床试验而引发争议。 FDA 工作人员和外部专家对该药物的有效性表示担忧。上个月公布的一项更大规模的试验结果显示，对患者的好处甚至不如抛硬币那么可靠。公司股价因此暴跌80%。

Approximately 6,000 individuals in the United States are diagnosed with ALS annually. The progressive disease gradually impairs the body's ability to move, speak, and eventually breathe, with no known cure.

美国每年约有 6,000 人被诊断患有 ALS。这种进行性疾病会逐渐损害身体的移动、说话和最终呼吸的能力，而且目前还没有已知的治愈方法。

Amylyx's decision to voluntarily withdraw Relyvrio from the market is an uncommon occurrence. At an FDA meeting in 2022, an expert panel member expressed skepticism about the prospect of removing the drug from the market if it proved ineffective, suggesting that the FDA had underestimated the complexity and difficulty of such an action.

Amylyx 决定自愿将 Relyvrio 从市场上撤回的情况并不常见。在 2022 年 FDA 的一次会议上，一位专家小组成员对如果证明该药物无效则将其从市场上撤下的前景表示怀疑，这表明 FDA 低估了此类行动的复杂性和难度。

"Frankly, I'm not sure it's ever taken place, although admittedly in rare cases, manufacturers themselves have made the decision to do so," commented Caleb Alexander, an internist at Johns Hopkins University.

约翰·霍普金斯大学的内科医生 Caleb Alexander 评论道：“坦率地说，我不确定这种情况是否曾经发生过，尽管在极少数情况下，制造商自己也做出了这样做的决定。”

Alexander commended Amylyx's decision on Thursday. "This case also serves as a reminder of our standards for market access and the risks associated with diluting these standards based on factors other than scientific evidence," he emphasized.

亚历山大周四赞扬了 Amylyx 的决定。他强调说：“这个案例也提醒我们，我们的市场准入标准以及基于科学证据以外的因素淡化这些标准所带来的风险。”

The ALS Association similarly applauded the announcement. "We commend Amylyx for withdrawing Relyvrio from the market while ensuring that people living with ALS can still access the drug if they believe it is beneficial," the organization stated.

ALS 协会同样对这一公告表示赞赏。该组织表示：“我们赞扬 Amylyx 将 Relyvrio 从市场上撤回，同时确保 ALS 患者在认为该药物有益的情况下仍然可以获得该药物。”

Amylyx, founded by Klee and Cohen while they were undergraduates at Brown University, had achieved significant success. The company gained FDA approval in 2022 based on a single trial indicating that Relyvrio slowed the rate of decline in activities such as walking and talking compared to a placebo. Subsequent data analysis also suggested that participants taking the drug lived an average of 10 months longer than a control group.

Amylyx 由 Klee 和 Cohen 在布朗大学读本科时创立，取得了巨大的成功。该公司于 2022 年获得 FDA 批准，基于一项试验表明，与安慰剂相比，Relyvrio 减缓了行走和说话等活动的下降速度。随后的数据分析还表明，服用该药物的参与者比对照组平均寿命长 10 个月。

Relyvrio generated approximately $380 million in revenue last year. At its peak, Amylyx boasted a market capitalization exceeding $1 billion.

Relyvrio 去年的收入约为 3.8 亿美元。在巅峰时期，Amylyx 的市值超过 10 亿美元。

Typically, the FDA requires more substantial evidence—two confirmatory trials—before approving a drug. However, the agency sometimes exercises flexibility, considering the severity of the disease and the efficacy of existing treatments.

通常，FDA 在批准药物之前需要更实质性的证据——两次验证性试验。然而，考虑到疾病的严重程度和现有治疗的疗效，该机构有时会采取灵活性。

Amylyx announced its intention to continue analyzing the results of its failed larger trial to inform future ALS research. The company is also developing an additional ALS drug, but its primary drug candidate currently targets Wolfram syndrome, a hereditary disorder that leads to diabetes.

Amylyx 宣布打算继续分析其失败的更大规模试验的结果，为未来的 ALS 研究提供信息。该公司还在开发另一种 ALS 药物，但其主要候选药物目前针对 Wolfram 综合征，这是一种导致糖尿病的遗传性疾病。

Correction:

更正：

An earlier version of this article incorrectly stated that Amylyx Pharmaceuticals made its announcement on Wednesday. The news was announced Thursday.

本文的早期版本错误地指出 Amylyx Pharmaceuticals 于周三发布了公告。该消息于周四宣布。