百濟神州(688235.SH)：百悦澤®用於治療華氏巨球蛋白血癥成人患者的新適應症上市申請獲得受理
格隆匯1月20日丨百濟神州(688235.SH)公佈，公司近日收到國家藥品監督管理局簽發的《受理通知書》，百悦澤®(澤布替尼膠囊)用於治療華氏巨球蛋白血癥成人患者的新適應症上市申請獲得受理。
百悦澤®是一款由公司自主研發的布魯頓氏酪氨酸激酶(BTK)小分子抑制劑，目前正在全球進行廣泛的臨牀試驗項目，作為單藥和與其他療法進行聯合用藥治療多種B細胞惡性腫瘤。由於新的BTK會在人體內不斷合成，百悦澤®的設計通過優化生物利用度、半衰期和選擇性，實現對BTK蛋白完全、持久的抑制。憑藉與其他獲批BTK抑制劑存在差異化的藥代動力學，百悦澤®能在多個疾病相關組織中抑制惡性B細胞增殖。
國家藥品監督管理局已於2021年6月批准百悦澤®用於治療R/RWM患者。此次新適應症上市申請有望將百悦澤®的適用範圍拓展至一線WM。
本次百悦澤®用於治療華氏巨球蛋白血癥成人患者的新適應症的上市申請是基於ASPEN試驗結果。ASPEN是一項隨機、開放標籤、多中心的3期臨牀試驗(NCT03053400)，對比了百悦澤®與伊布替尼用於治療復發/難治性(R/R)或初治(TN) WM患者的數據。
經獨立審查委員會根據第六屆華氏巨球蛋白血癥國際研討會修訂版緩解標準(Treon 2015)評估，百悦澤®治療組在總體意向性治療人羣中的完全緩解(CR)和非常好的部分緩解(VGPR)率為28%(95%置信區間：20，38)，而伊布替尼組為19%(95%置信區間：12，28)。儘管兩組數據的差異未達到統計學顯著性(p值=0.09)，但百悦澤®治療組展現了更高的VGPR率數據，且緩解質量呈現出改善趨勢。
在 ASPEN試驗中，與伊布替尼相比，百悦澤®取得了更具優勢的安全性數據，特定不良事件的發生率更低，包括房顫/房撲(百悦澤®為2%對比伊布替尼15%)和大出血(百悦澤®為6%對比伊布替尼 9%)。在101例接受百悦澤®治療的WM患者中，4%的患者因不良事件而終止治療，14%的患者因不良事件降低用藥劑量。
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