Amylyx Pharmaceuticals 在臨床試驗失敗後撤回 ALS 藥物

Amylyx Pharmaceuticals 宣布裁員 70%，並在臨床試驗顯示其 ALS 藥物 Relyvrio/Albrioza 無效後自願撤回該藥物。此舉是在美國食品和藥物管理局基於一項試驗對該藥物進行有爭議的批准之後發生的。該公司已與監管機構啟動移除程序，並將在過渡期間向現有患者提供免費藥物取得。這項決定很不尋常，因為藥物從市場上撤下通常是不常見的。

Amylyx Pharmaceuticals Withdraws ALS Drug from Market

Amylyx Pharmaceuticals 從市場上撤回 ALS 藥物

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 宣布決定自願將其藥物 Relyvrio（加拿大 Albrioza）撤出市場，這是一項重大進展。這項措施是在一項臨床試驗發布後不到一個月做出的，該試驗表明曾經有希望的肌萎縮側索硬化症（ALS）（也稱為盧伽雷氏症）治療方法無效。

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.

這家總部位於馬薩諸塞州劍橋的公司週四與監管機構啟動了撤回該藥物的程序。立即生效，Relyvrio 將不再對新患者開放。然而，現有患者可以選擇過渡到「免費藥物計劃」。 Amylyx 也將裁員 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.

聯合執行長約書亞·科恩(Joshua Cohen) 和賈斯汀·克利(Justin Klee) 表示：「雖然對於ALS 社區來說這是一個充滿挑戰的時期，但我們與受影響的利益相關者合作，並遵循我們對ALS 和其他神經退化性疾病患者堅定不移的承諾，共同邁出了這條道路。」Amylyx 的執行官在聯合聲明中表示。

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.

美國食品藥物管理局 (FDA) 於 2022 年批准 Relyvrio，因其依賴單一臨床試驗而引發爭議。 FDA 工作人員和外部專家對該藥物的有效性表示擔憂。上個月公佈的一項更大規模的試驗結果顯示，對患者的好處甚至不如拋硬幣那麼可靠。公司股價因此暴跌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.

美國每年約有 6,000 人被診斷出患有 ALS。這種進行性疾病會逐漸損害身體的移動、說話和最終呼吸的能力，而且目前還沒有已知的治癒方法。

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 決定自願將 Relyvrio 從市場上撤回的情況並不常見。在 2022 年 FDA 的一次會議上，一位專家小組成員對如果證明該藥物無效則將其從市場上撤下的前景表示懷疑，這表明 FDA 低估了此類行動的複雜性和難度。

"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.

約翰霍普金斯大學的內科醫生Caleb Alexander 評論道：「坦白說，我不確定這種情況是否曾經發生過，儘管在極少數情況下，製造商自己也做出了這樣做的決定。 」

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.

亞歷山大周四讚揚了 Amylyx 的決定。他強調：“這個案例也提醒我們，我們的市場准入標準以及基於科學證據以外的因素淡化這些標準所帶來的風險。”

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 協會也對此公告表示讚賞。該組織表示：“我們讚揚 Amylyx 將 Relyvrio 從市場上撤回，同時確保 ALS 患者在認為該藥物有益的情況下仍然可以獲得該藥物。”

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 在布朗大學攻讀本科時創立，取得了巨大的成功。該公司於 2022 年獲得 FDA 批准，基於一項試驗表明，與安慰劑相比，Relyvrio 減緩了行走和說話等活動的下降速度。隨後的數據分析還表明，服用該藥物的參與者比對照組平均壽命長 10 個月。

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

Relyvrio 去年的營收約為 3.8 億美元。在巔峰時期，Amylyx 的市值超過 10 億美元。

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 在批准藥物之前需要更實質的證據—兩次驗證性試驗。然而，考慮到疾病的嚴重程度和現有治療的療效，該機構有時會採取靈活性。

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 宣布打算繼續分析其失敗的更大規模試驗的結果，為未來的 ALS 研究提供資訊。該公司還在開發另一種 ALS 藥物，但其主要候選藥物目前針對 Wolfram 綜合徵，這是一種導致糖尿病的遺傳性疾病。

Correction:

更正：

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

本文的早期版本錯誤地指出 Amylyx Pharmaceuticals 於週三發布了公告。該消息於週四宣布。