The U.S. Food and Drug Administration (FDA) has changed its position on how it will approve AMT-130, an experimental treatment for Huntington's disease developed by uniQure.

美國食品藥品監督局（FDA）改變了對於 uniQure 開發的亨丁頓舞蹈症（Huntington's disease）實驗性治療方案 AMT-130 的審批立場。

This new agreement follows a period of disagreement between the company and the agency. In March, the FDA described the therapy as a failed product because there was not enough data. The agency had previously demanded a new clinical trial using a 'sham surgery' as a control, which many people criticized. However, the FDA has now agreed that a three-year analysis from an earlier stage can be used as the main evidence for a license application. Consequently, uniQure can now seek accelerated approval and plans to submit its application in the third quarter of 2026.

這項新協議是在公司與該機構經歷一段時間的分歧後達成的。三月份，FDA 因為數據不足而將該療法描述為失敗產品。該機構先前要求進行一項使用「偽手術」作為對照組的新臨床試驗，這引起了許多人的批評。然而，FDA 現在同意可以使用早期階段的三年分析作為申請許可的主要證據。因此，uniQure 現在可以尋求加速審批，並計劃在 2026 年第三季提交申請。

There are different views on this decision. The Huntington’s Disease Society of America (HDSA) and other patient groups, who had previously sent petitions to Congress, described the move as a major success. On the other hand, medical experts emphasized that final approval still depends on further studies to prove the treatment works over the long term. Furthermore, the Department of Health and Human Services (HHS) asserted that this approach balances the urgent need for new treatments with the requirement for high scientific standards.

對於這項決定，外界看法不一。美國亨丁頓舞蹈症協會（HDSA）及其他先前向國會遞交請願書的患者團體，將此舉描述為一次重大成功。另一方面，醫學專家強調，最終審批仍取決於進一步的研究，以證明該療法在長期內有效。此外，衛生及公共服務部（HHS） asserted 稱，這種方法在對新療法迫切的需求與對高科學標準的要求之間取得了平衡。

This change is part of a larger trend of inconsistent rules. Similar shifts in requirements have happened with Moderna’s flu vaccine and Sarepta’s Elevidys. These changes have caused concerns about whether the FDA is consistent when overseeing new biotechnologies. Meanwhile, the stock market reacted positively to the news, and uniQure's share price rose by more than 75 percent.

這次變動是規則不一致之大趨勢的一部分。類似的要求轉變也發生在 Moderna 的流感疫苗和 Sarepta 的 Elevidys 上。這些變化引發了人們對 FDA 在監管新生物技術時是否保持一致的擔憂。與此同時，股市對此消息反應正面，uniQure 的股價上漲超過 75%。

The FDA has created a possible path for AMT-130 to be approved, although the final decision depends on future data.

FDA 為 AMT-130 創造了一條可能的獲批路徑，儘管最終決定仍取決於未來的數據。