FDA 核准治療罕見亨特氏症的新療法
FDA Approves New Treatment for Rare Hunter Syndrome
2026年3月25日，美國食品藥物管理局（FDA）批准了一項名為Avlayah（tividenofusp alfa-eknm）的新療法，用於治療罕見遺傳疾病——亨特氏症（Hunter syndrome）。
On March 25, 2026, the FDA granted accelerated approval to a new treatment called Avlayah (tividenofusp alfa-eknm) for Hunter syndrome, a rare genetic disorder.
亨特氏症主要影響男性，其病理特徵是細胞內有害物質堆積，進而導致嚴重的認知與運動功能衰退。
Hunter syndrome, which mostly affects males, involves a dangerous buildup of substances in cells that leads to severe cognitive and motor decline.
過去的療法無法穿透大腦，但Avlayah採用了特殊的傳輸技術，專門設計用於穿過血腦屏障（Blood-brain barrier）。
Previously available therapies could not reach the brain, but Avlayah is specifically engineered to cross the blood-brain barrier using a special delivery technology.
FDA授予加速批准（Accelerated approval）的決定，是基於腦脊髓液中特定標記的減少，此舉突顯了FDA在面對罕見疾病時，願意採取替代終點（Surrogate endpoints）的作法。
The FDA's decision to grant accelerated approval was based on a reduction of certain markers in the cerebrospinal fluid, a move that highlights the agency's willingness to use surrogate endpoints when dealing with rare diseases.
