Pfizer Says U.S. FDA Approved Company's BEQVEZ (Fidanacogene Elaparvovec-dzkt), A One-Time Gene Therapy For Adults With Hemophilia B; Company Now Plans Launching An Innovative Warranty Program Based On Durability Of Patient Response To Treatment
Pfizer Says U.S. FDA Approved Company's BEQVEZ (Fidanacogene Elaparvovec-dzkt), A One-Time Gene Therapy For Adults With Hemophilia B; Company Now Plans Launching An Innovative Warranty Program Based On Durability Of Patient Response To Treatment
With BEQVEZ now approved for use, Pfizer is launching an innovative warranty program based on durability of patient response to treatment. The goal of the warranty is to provide greater certainty to payers, maximize access for eligible patients who receive BEQVEZ, and offer financial protection by insuring against the risk of efficacy failure.
隨着BEQVEZ現已獲准使用，輝瑞正在推出一項創新的保修計劃，該計劃基於患者對治療的耐久性。保修的目標是爲付款人提供更大的確定性，最大限度地爲符合條件的接受BEQVEZ的患者提供機會，並通過爲療效失效風險投保來提供經濟保障。
"For people living with hemophilia, disease management can interfere with many aspects of their lives. A one-time infusion of BEQVEZ may allow eligible patients more time for the things they love," said Kim Phelan, Chief Operating Officer, The Coalition for Hemophilia B. "We are excited to have BEQVEZ as a promising treatment option for eligible people living with hemophilia B. We look forward to learning more and celebrating with the community and with Pfizer at our annual conference that is currently taking place."
“對於血友病患者來說，疾病管理會干擾他們生活的許多方面。一次性注射BEQVEZ可以讓符合條件的患者有更多時間去做自己喜歡的東西，” 乙型血友病聯盟首席運營官金·費蘭說，“我們很高興BEQVEZ成爲符合條件的乙型血友病患者的一種有前途的治療選擇。我們期待在目前舉行的年度會議上與社區和輝瑞一起了解更多，並與輝瑞一起慶祝。”
BEQVEZ is currently under review with the European Medicines Agency (EMA), and the treatment recently received regulatory approval in Canada. In addition to BEQVEZ, Pfizer currently has two other Phase 3 programs investigating gene therapy in populations where there is a high unmet need: hemophilia A (giroctocogene fitelparvovec) and Duchenne muscular dystrophy (fordadistrogene movaparvovec). Additionally, a Phase 3 trial is investigating marstacimab, a novel, investigational anti-tissue factor pathway inhibitor for the treatment of people with hemophilia A and B with and without inhibitors. A Biologics License Application and European Marketing Authorization Application for marstacimab are currently under review with the FDA and EMA, respectively.
BEQVEZ目前正在接受歐洲藥品管理局（EMA）的審查，該療法最近獲得了加拿大的監管批准。除BEQVEZ外，輝瑞目前還有另外兩個三期計劃在需求未得到滿足的人群中研究基因療法：甲型血友病（giroctocogene fitelparvovec）和杜興氏肌肉萎縮症（fordadistrogene movaparvovec）。此外，一項3期試驗正在研究marstacimab，這是一種新的在研抗組織因子途徑抑制劑，用於治療有無抑制劑的A型和B型血友病患者。目前，FDA和EMA正在分別審查馬斯塔西單抗的生物製劑許可證申請和歐洲上市許可申請。
譯文內容由第三人軟體翻譯。