近日，美国FDA批准Repatha（evolocumab）注射剂用于某些使用目前治疗选择不能使其低密度脂蛋白胆固醇得到控制的患者。
Repatha是新型 PCSK9 抑制剂类药物中第二款获得批准的药物，该药物被批准结合饮食及最大耐受量的他汀药物用于需要额外降低LDL胆固醇的杂合子家族性高胆固醇血症（HeFH）、纯合子家族性高胆固醇血症（HoFH）、或临床上有动脉粥样硬化性心血管病，如心脏病发作或中风的患者。
家族性高胆固醇血症（包括 HeFH 和 HoFH）是一种遗传性疾病，它可以导致高水平的LDL胆固醇。血液中高水平LDL胆固醇与心血管或心脏疾病相关。心脏疾病是美国男女患者死亡的第一杀手。据美国疾病控制与预防中心提供的信息，美国每年大约有61万人死于心脏疾病，这相当于每4例死亡中就有1例是因心脏疾病死亡。
药品评价和研究中心新药室主任说：“Repatha提供在这个新类型另外治疗选择为患者有家族性超胆固醇血症或已知心血管病用他汀类不足以减低其LDL胆固醇，” “心血管病是严重威胁美国人健康，和FDA承诺促进发展和批准有效和安全药物解决这个重要的公共健康问题。”
批准日期：2015年8月27日；公司：Amgen Inc.
HIGHLIGHTS OF PRESCRIBING INFORMATION
These highlights do not include all the information needed to use REPATHA ® safely and effectively. See full prescribing information for REPATHA .
REPATHA (evolocumab) injection, for subcutaneous use
Initial U.S. Approval: 2015
RECENT MAJOR CHANGES
Dosage and Administration (2.2) 7/2016
INDICATIONS AND USAGE
REPATHA is a PCSK9 (proprotein convertase subtilisin kexin type9) inhibitor antibody indicated as an adjunct to diet and:
Maximally tolerated statin therapy for treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (CVD), who require additional lowering of low density lipoprotein cholesterol (LDL-C). (1.1)
Other LDL-lowering therapies (e.g., statins, ezetimibe, LDL apheresis) in patients with homozygous familial hypercholesterolemia (HoFH) who require additional lowering of LDL-C. (1.2)
Limitations of Use
The effect of REPATHA on cardiovascular morbidity and mortality has not been determined. (1.3)
DOSAGE AND ADMINISTRATION
Administer subcutaneously. (2.1)
Primary hyperlipidemia with established clinical atherosclerotic CVD or HeFH: 140 mg every 2 weeks or 420 mg once monthly in abdomen, thigh, or upper arm. (2.1)
HoFH: 420 mg once monthly. (2.1)
The 420 mg dose of REPATHA can be administered:
○ over 9 minutes by using the single-use on-body infusor with prefilled cartridge, or
○ by giving 3 injections consecutively within 30 minutes using the single-use prefilled autoinjector or single-use prefilled syringe. (2.2)
See Dosage and Administration for important administration instructions. (2.2)
DOSAGE FORMS AND STRENGTHS
Injection: 140 mg/mL solution in a single-use prefilled syringe (3)
Injection: 140 mg/mL solution in a single-use prefilled SureClick® autoinjector (3)
Injection: 420 mg/3.5 mL solution in a single-use PushtronexTM system (on-body infusor with prefilled cartridge) (3)
CONTRAINDICATIONS
Patients with a history of a serious hypersensitivity reaction to REPATHA. (4)
WARNINGS AND PRECAUTIONS
Allergic Reactions: Rash and urticaria have occurred. If signs or symptoms of serious allergic reactions occur, discontinue treatment with REPATHA, treat according to the standard of care, and monitor until signs and symptoms resolve. (5.1)
ADVERSE REACTIONS
Common adverse reactions in clinical trials (> 5% of patients treated with REPATHA and occurring more frequently than placebo): nasopharyngitis, upper respiratory tract infection, influenza, back pain, and injection site reactions. (6)
To report SUSPECTED ADVERSE REACTIONS, contact Amgen Medical Information at 1-800-77-AMGEN (1-800-772-6436) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
See 17 for PATIE