2016年5月31日，杰特贝林制药宣布，美国食品药品监督管理局（FDA）已批准Afstyla（抗血友病因子[重组]，单链；CSL-Behring）用于成人和儿童血友病A的常规预防，以降低出血发生的频率。Afstyla是一种新型、持久的重组因子VIII单链疗法，旨在提高分子稳定性和延长作用时间。
该批准是基于AFFINITY临床开发项目的结果，该项目包括两项关键和一项扩展的开放标签多中心研究，评估阿夫Styla在成人、青少年和儿童血友病A中的安全性和有效性。该项目的数据显示，成人和儿童研究的中位年自发出血率（AsBR）均为0.00。对于预防性使用Afstyla的患者，成人和青少年的中位年出血率（ABR）为1.14，12岁以下儿童为3.69。
在这项关键的试验中，每周输注不超过两次的Afstyla可以有效控制94%的成人和青少年出血以及96%的儿科患者出血。仅一次输注就有效控制了81%的成人和青少年出血以及86%的儿童出血。
试验期间报告的最常见不良反应是头晕和超敏反应。Afstyla显示出强大的安全性，未观察到抑制剂。
Afstyla预计将于今年夏初上市。它将装在一个试剂盒中供应，该试剂盒含有冻干粉，装在一次性小瓶中，标称含有250、500、1000、2000或3000国际单位（IU）。
信息来源：https://www.rarediseaseadvisor.com/therapies/afstyla-antihemophilic-factor-recombinant-single-chain/
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AFSTYLA GENERIC NAME & FORMULATIONS
Legal Class
Rx
General Description
Antihemophilic Factor (recombinant) single chain 250 IU, 500 IU, 1000 IU, 2000 IU, 3000 IU; per vial; lyophilized pwd for IV infusion after reconstitution; preservative-free.
Pharmacological Class
Clotting factor.
How Supplied
Kit—1(vial w.diluent+supplies)
Manufacturer
CSL Behring, LLC
Generic Availability
NO
AFSTYLA INDICATIONS
Indications
In patients with Hemophilia A: to treat and control bleeding episodes, for perioperative management, and routine prophylaxis to reduce the frequency of bleeding episodes.
Limitations of Use
Not for treating von Willebrand disease.
AFSTYLA DOSAGE AND ADMINISTRATION
Adults and Children
Dosage Required (IU) = Body Weight (kg) × Desired % Factor VIII Increase × 0.5. Individualize. Max infusion rate: 10mL/min. Bleeding: Minor: obtain 20–40% FVIII increase; may repeat every 12–24hrs until resolved; Moderate: obtain 30–60% FVIII increase; may repeat every 12–24hrs until resolved; Major: obtain 60–100% FVIII increase; may repeat every 8–24hrs until resolved. Perioperative: Minor: obtain 30–60% FVIII increase; may repeat every 24hrs for ≥1 day until healed; Major: obtain 80–100% FVIII increase; may repeat every 8–24hrs until adequately healed, then continue for ≥7 days to maintain Factor VIII activity of 30–60%. Routine prophylaxis: ≥12yrs: 20–50 IU/kg 2–3 times weekly; <12yrs: 30–50 IU/kg 2–3 times weekly (more frequent or higher doses may be required).
AFSTYLA CONTRAINDICATIONS
Contraindications
Hamster protein sensitivity.
AFSTYLA BOXED WARNINGS
Not Applicable
AFSTYLA WARNINGS/PRECAUTIONS
Warnings/Precautions
Confirm Factor VIII deficiency prior to treatment. Monitor for development of Factor VIII inhibitors. Discontinue if hypersensitivity reactions occur; consider premedication with antihistamines if previous history of hypersensitivity. Pregnancy. Nursing mothers.
AFSTYLA PHARMACOKINETICS
See Literature
AFSTYLA INTERACTIONS
Not Applicable
AFSTYLA ADVERSE REACTIONS
Adverse Reactions
Dizziness, hypersensitivity, paresthesia, rash, erythema, pruritus, pyrexia, inj site pain, chills, feeling hot.
AFSTYLA CLINICAL TRIALS
See Literature
AFSTYLA NOTE
Not Applicable
AFSTYLA PATIENT COUNSELING
See Literature |
