Vertex Announces New Drug Submission For Exagamglogene Autotemcel Has Been Accepted For Priority Review By Health Canada For The Treatment Of Sickle Cell Disease And Transfusion-Dependent Beta Thalassemia
Vertex Announces New Drug Submission For Exagamglogene Autotemcel Has Been Accepted For Priority Review By Health Canada For The Treatment Of Sickle Cell Disease And Transfusion-Dependent Beta Thalassemia
The NDS will be part of an aligned review with Health Technology Assessment (HTA) organizations, the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut national d'excellence en santé et en services sociaux (INESSS) in Quebec.-Exa-cel is the first CRISPR-based gene-edited therapy to be submitted for Health Canada review-
-exa-cel 是第一種提交加拿大衛生部審查的基因編輯療法-
TORONTO, April 1, 2024 /CNW/ - Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced its New Drug Submission (NDS) for exagamglogene autotemcel (exa-cel) has been accepted for Priority Review by Health Canada for the treatment of patients aged 12 years and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) and for the treatment of patients aged 12 years and older with transfusion-dependent beta thalassemia (TDT).
多倫多，2024年4月1日 /CNW/-Vertex Pharmicals Incorporatem（納斯達克股票代碼：VRTX）今天宣佈，其exagamglogene autotemcel（exa-cel）的新藥申請（NDS）已被加拿大衛生部接受優先審查，用於治療復發血管閉塞危象（VOC）的12歲及以上鐮狀細胞病（SCD）患者，以及治療12歲患者及年齡較大的人患有輸血依賴性 β 地中海貧血 (TDT)。
"We are pleased that exa-cel has been accepted for Priority Review by Health Canada and look forward to bringing this therapy to eligible patients," said Michael Siauw, General Manager at Vertex Pharmaceuticals (Canada) Incorporated.
Vertex Pharmicals（加拿大）公司總經理邁克爾·西亞烏說：“我們很高興exa-cel被加拿大衛生部接受優先審查，並期待將這種療法帶給符合條件的患者。”
The NDS will be part of an aligned review with Health Technology Assessment (HTA) organizations, the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut national d'excellence en santé et en services sociaux (INESSS) in Quebec.
NDS將是與衛生技術評估（HTA）組織、加拿大衛生藥物和技術局（CADTH）和魁北克國家健康和社會服務卓越研究所（INESSS）共同審查的一部分。
With Priority Review, the conventional review timeline of 300 days is reduced to 180 days.
通過優先審核，將傳統的 300 天審核期限縮短爲 180 天。
The NDS is supported by results from the ongoing Phase 3 studies, CLIMB-111 and CLIMB-121, as well as an ongoing long-term follow-up study, CLIMB-131. Data from the Phase 3 studies were most recently presented at the American Society of Hematology (ASH) Annual Meeting and Exposition in December of 2023.
NDS得到了正在進行的3期研究（CLIMB-111 和 CLIMB-121）以及正在進行的長期隨訪研究 CLIMB-131 的結果的支持。3期研究的數據最近在2023年12月的美國血液學會（ASH）年會和博覽會上公佈。
譯文內容由第三人軟體翻譯。