FDA Green Lights Pfizer's Gene Therapy For Rare Bleeding Disorder Hemophilia
FDA Green Lights Pfizer's Gene Therapy For Rare Bleeding Disorder Hemophilia
Friday, the FDA approved Pfizer Inc's (NYSE:PFE) Beqvez (fidanacogene elaparvovec-dzkt) for moderate to severe hemophilia B in adult patients who currently use factor IX (FIX) prophylaxis therapy or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var capsid.
週五，美國食品藥品管理局批准輝瑞公司（紐約證券交易所代碼：PFE）Beqvez（fidanacogene elaparvovec-dzkt）治療中度至重度乙型血友病，這些患者目前正在使用IX因子（FIX）預防療法，或者目前或歷史上有危及生命的出血，或者反覆出現嚴重的自發性出血發作，並且沒有針對腺相關病毒血清的中和抗體輸入 rh74var 衣殼。
Beqvez is a one-time treatment designed to enable hemophilia B patients to produce FIX themselves rather than the current standard of care, which requires regular intravenous infusions of FIX that are often administered multiple times a week or multiple times a month.
Beqvez是一種一次性治療方法，旨在使乙型血友病患者能夠自己進行FIX治療，而不是目前的護理標準，後者需要定期靜脈注射FIX，通常每週多次或每月多次給藥。
Hemophilia B is a rare genetic bleeding disorder that prevents normal blood clotting because of a deficiency in FIX that causes those with the disease to bleed more frequently and longer than others.
乙型血友病是一種罕見的遺傳性出血性疾病，由於缺乏 FIX，會導致該病患者比其他人更頻繁和更長時間地流血，從而阻止血液的正常凝結。
The approval is based on the BENEGENE-2 Phase 3A study, which showed that a single dose of Pfizer's therapy helped eliminate bleeding in 60% of patients compared to 29% in the prophylaxis arm.
該批准基於 BENEGENE-2 3A 期研究，該研究表明，輝瑞的單劑量療法有助於消除60％的患者的出血，而預防組的這一比例爲29％。
A median annualized bleeding rate (ABR) of zero (range of 0 to 19) was observed during the efficacy evaluation period compared to the prophylaxis arm, in which a median ABR of 1.3 (range of 0 to 53.9) was observed.
與預防組相比，在療效評估期間，觀察到的年化出血率中位數（ABR）爲零（範圍在0至19之間），在預防組中，觀察到的中位ABR爲1.3（範圍在0至53.9之間）。
Pfizer said it will continue to monitor the long-term durability and safety of the treatment over 15 years.
輝瑞表示，它將在15年內繼續監測該治療的長期耐久性和安全性。
With Beqvez now approved for use, Pfizer is launching a warranty program based on the durability of patient response to treatment.
隨着Beqvez現已獲准使用，輝瑞正在啓動一項基於患者對治療反應的耐久性的保修計劃。
The warranty aims 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.
保修旨在爲付款人提供更大的確定性，最大限度地爲接受Beqvez的符合條件的患者提供便利，並通過爲療效失效風險投保來提供財務保障。
Beqvez is currently under review with the European Medicines Agency, and the treatment recently received regulatory approval in Canada.
Beqvez目前正在接受歐洲藥品管理局的審查，該療法最近獲得了加拿大的監管批准。
Reuters noted that Pfizer has set a list price for Beqvez at $3.5 million in the U.S., similar to Australian drugmaker CSL Ltd's (OTC:CSLLY) rival gene therapy Hemgenix.
路透社指出，輝瑞已將Beqvez在美國的標價定爲350萬美元，與澳大利亞製藥商CSL Ltd（場外交易代碼：CSLLY）的競爭對手基因療法Hemgenix類似。
Price Action: PFE shares are up 0.88% at $25.48 at the last check Friday.
價格走勢：週五最後一次檢查時，PFE股價上漲0.88％，至25.48美元。
Photo via Shutterstock
照片來自 Shutterstock
譯文內容由第三人軟體翻譯。