FDA Approves Pfizer's Second Hemophilia Drug With Six Months
FDA Approves Pfizer's Second Hemophilia Drug With Six Months
Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (FVIII in hemophilia A, FIX in hemophilia B), impacting more than 800,000 people globally.On Friday, the FDA approved Pfizer Inc's (NYSE:PFE) Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients with hemophilia A (congenital factor VIII deficiency), or hemophilia B (congenital factor IX deficiency).
上週五，FDA批准了輝瑞公司（紐交所:PFE）的Hympavzi（馬斯他西單抗-hncq），用於預防或減少血友病A（先天性凝血因子Ⅷ缺乏症）或血友病B（先天性凝血因子Ⅸ缺乏症）成人和小兒患者出血發作的常規預防。
Hympavzi is the first and only anti-tissue factor pathway inhibitor (anti-TFPI) approved in the U.S. for hemophilia A or B and the first hemophilia medicine approved in the U.S. to be administered via a pre-filled, auto-injector pen.
Hympavzi是美國首個且唯一獲批用於血友病A或B的抗組織因子途徑抑制劑（TFPI抑制劑），也是美國首個獲批通過預裝自動注射器筆進行給藥的血友病藥物。
Hympavzi can offer a subcutaneous treatment option with a once-weekly dosing schedule and minimal preparation required for each individual administration.
Hympavzi可以提供皮下治療選擇，每週一次劑量安排，每次個體給藥所需的準備工作很少。
Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (FVIII in hemophilia A, FIX in hemophilia B), impacting more than 800,000 people globally.
血友病是一類罕見的由凝血因子缺乏（血友病A爲FVIII，血友病B爲FIX）引起的家族性遺傳性血液疾病，全球影響超過800,000人。
Results from the Phase 3 BASIS trial (NCT03938792) supported the FDA approval of Hympavzi.
來自3期BASIS試驗（NCT03938792）的結果支持了Hympavzi的FDA批准。
In the study, Hympavzi reduced the annualized bleeding rate (ABR) for treated bleeds by 35% and 92% after a 12-month active treatment period compared to routine prophylaxis (RP) and on-demand (OD) treatment, respectively, in patients with hemophilia A or B without inhibitors.
在研究中，Hympavzi相對於例行預防和按需治療分別使血友病A或B無抑制劑患者在經過12個月的積極治療後，治療出血的年化出血率（ABR）分別減少了35%和92%。
The Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion for marstacimab for the routine prophylaxis of bleeding episodes in adults and adolescents with severe hemophilia A or hemophilia B.
歐洲藥品管理局人用藥品委員會對馬斯他西單抗在成年人和青少年中用於血友病A或B的常規預防採納了積極意見。
Reuters, citing some analysts, highlights that Hympavzi sales are expected to reach $300 million by 2030.
路透社援引一些分析師的話稱，預計Hympavzi的銷售額將在2030年達到3億美元。
Earlier this year, FDA approved Pfizer's 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.
今年早些時候，FDA批准了輝瑞的Beqvez（fidanacogene elaparvovec-dzkt），用於目前使用因子IX（FIX）預防治療的成年患者中度至重度血友病b，或有當前或歷史上有生命威脅的出血，或有反覆、嚴重的自發性出血發作，並且沒有對腺相關病毒血清型Rh74var殼蛋白出現中和抗體的患者。
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，旨在使血友病b患者能夠自行產生FIX，而不是當前的標準護理，該護理需要定期靜脈輸注FIX，通常每週多次或每月多次。
In July, Pfizer, in collaboration with Sangamo Therapeutics Inc, released results from the Phase 3 AFFINE study of giroctocogene fitelparvovec, an investigational gene therapy for adult patients with moderately severe to severe hemophilia A.
在7月，輝瑞與Sangamo Therapeutics Inc合作，發佈了對於中度嚴重至重度血友病A成年患者的研究基因治療試驗Phase 3 AFFINE研究的giroctocogene fitelparvovec的結果。
Price Action: PFE stock is down 0.14% at $29.12 during the premarket session at last check Monday.
股價走勢：PFE股票在上週一最後一次查看盤前交易時下跌0.14%，報29.12美元。
Photo via Shutterstock
圖片來自shutterstock。
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譯文內容由第三人軟體翻譯。
