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2015年12月8日,FDA批准Baxalta的Vonvendi（重组血管性血友病因子）， 用于18岁及以上的成年人，治疗血管性血友病（VWD）。Vonvendi是FDA第一个被批准的重组血管性血友病因子，批准用于诊断出有VWD的成年患者有需要治疗或控制出血。
VWD是最常见的遗传性出血性疾病，影响大约1%的美国人。男女患VWD概率一样，是由一种血管性血友病因子的缺乏或缺陷引起的，而这种蛋白质，是正常血液凝固的关键。患有VWD的患者可能会发展成为严重的出血，可以从鼻子、牙龈和肠道，以及肌肉和关节处。患有VWD的女性月经可能经持续时间超过平均值，在分娩后可能出血过多。
FDA生物制品评价和研究中心主任Karen Midthun，医学博士，说：‘遗传性出血性疾病患者应与医疗护理提供者讨论适当的措施，以减少失血，Vonvendi 的批准为患者提供一个额外的治疗选择，用于治疗的血友病患者出血。
Vonvendi的疗效及安全性评价通过两项临床试验，69位VWD成人参与。这些试验证明，Vonvendi是安全、有效，按需治疗和控制从身体的各种不同部位出血。在临床试验中，没有安全问题需要担心。观察到的最常见的不良反应就是广义的瘙痒。
VONVENDI[von Willebrand factor (Recombinant)]
Is in your cornerA recombinant von Willebrand factor with an option to dose independently of Recombinant Factor VIII For each bleeding episode, administer the first dose of VONVENDI with an approved recombinant (non-von Willebrand factor-containing) factor VIII (rFVIII) if FVIII baseline levels are <40% or are unknown. VONVENDI can be administered without rFVIII in some patients if FVIII levels are ≥40% normal activity (0.4 IU/mL) or if an urgent increase is not needed。
VONVENDI Rx
Generic Name and Formulations:
Von Willebrand Factor (recombinant); 650 IU VWF:RCo, 1300 IU VWF:RCo; per vial; lyophilized pwd for IV inj after reconstitution; preservative-free.
Company:
Baxalta
Select therapeutic use: Bleeding disorders
RECENT UPDATES Monograph added.
Indications for VONVENDI:
On-demand treatment and control of bleeding episodes in adults with von Willebrand disease (VWD).
Adult:
Give recombinant factor VIII (FVIII) with first infusion if baseline plasma FVIII level <40% or is unknown (see full labeling). ≥18yrs: Minor bleed: initially 40–50 IU/kg, then every 8–24 hours as needed. Major bleed: initially 50–80 IU/kg, then 40–60 IU/kg every 8–24 hours for 2–3 days as needed. Monitor and adjust according to extent and location of bleed. Max infusion rate: 4mL/min.
Children:
<18yrs: not established.
Contraindications:
Hypersensitivity to hamster or mouse proteins.
Warnings/Precautions:
Treatment should be supervised by physician. Risk of thrombotic events in patients with known risk factors or an excessive rise in FVIII levels; monitor. Discontinue immediately if severe allergic reactions occur. Ineffectiveness may indicate antibody formation; monitor and consider alternative therapy. Pregnancy. Nursing mothers.
Pharmacological Class:
Clotting factor.
Adverse Reactions:
Generalized pruritus; antibody formation.
Generic Availability:
NO
How Supplied:
Single-use vials—1 (w. diluent, supplies) | 
