2014年10月22日讯 /生物谷BIOON/ --百特国际（Baxter International）和合作伙伴Halozyme公司近日联合宣布，在美国推出皮下注射（SC）药物HyQvia，该药于今年9月获FDA批准，用于原发性免疫缺陷症（PI）的治疗。HyQvia由人正常免疫球蛋白（IG，10%）和重组人透明质酸酶（hyaluronidase）组成，透明质酸酶有利于IG在皮下的分散和吸收。
HyQvia是获批用于原发性免疫缺陷症（PI）治疗的首个每月一次的皮下注射（subcutaneous，SC）免疫球蛋白（IG），每月仅需皮下注射一次，且在单个注射部位即可递送全部的治疗剂量。目前，大多数PI患者需在医院接受静脉输液治疗，而当前的皮下注射IG药物，需要每周或每2周注射一次，且每次注射时需在多个位点注射。
2年前的2012年8月，FDA因安全性问题拒绝批准HyQvia，FDA提及的问题包括非中和抗体（non-neutralizing antibodies）对生殖、发育及生育可能产生的潜在影响。非中和抗体是指能够与目标蛋白结合的的抗体，但所结合区域与蛋白功能无关。之后，百特和Halozyme应FDA要求，提交了额外的临床前数据，并于2013年12月提交了有关HyQvia的一份修订版生物制品许可申请（BLA），重新启动关于HyQvia用于原发性免疫缺陷（PI）成人患者治疗的审查程序。
原有BLA的提交，基于一项前瞻性、开放标签、非对照、多中心III期临床试验的数据。该项研究评估了HyQvia用于预防急性严重细菌感染的安全性和有效性，同时与静脉给药型免疫球蛋白的药代动力学参数进行了比较。该项研究的目的是在一处皮下注射位点注入3或4周剂量的HyQvia。该项研究中，急性严重细菌感染率为0.025/人/年，低于所要求的疗效阈值1.0/人/年。对HyQvia的耐受性评估数据表明，最常见的不良反应为输注部位反应、头痛、疲劳、发热。
此前，HyQvia已于2013年5月获欧盟批准，作为一种替代疗法，用于原发性免疫缺陷综合征（primary immunodeficiency syndromes）及伴有继发性低丙种球蛋白血症和反复感染的骨髓瘤或慢性淋巴细胞白血病成人患者（≥18岁）的治疗。该药已在数个欧洲国家上市，包括德国、荷兰、瑞典、挪威、丹麦、爱尔兰和意大利。
英文原文：Baxter Launches HYQVIA in the United States for Adult Patients with Primary Immunodeficiency
DEERFIELD, Ill., October 20, 2014 - Baxter International Inc. (NYSE:BAX) and Halozyme Therapeutics, Inc., (NASDAQ:HALO) today announced the launch and first shipments of HYQVIA [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase], Baxter's subcutaneous immunoglobulin treatment for adult patients with primary immunodeficiency (PI) in the United States. The treatment was approved by the U.S. Food and Drug Administration (FDA) in September of this year.
"HYQVIA will have a significant impact on the treatment of primary immunodeficiency. It will be a welcomed addition to the therapeutic options in my practice, particularly for patients who are seeking less frequent dosing, but still desire the efficacy and tolerability associated with subcutaneous treatment," said Richard L. Wasserman, M.D., Ph.D., clinical professor of pediatrics at University of Texas Southwestern Medical School and clinical investigator on the HYQVIA clinical trial.
HYQVIA is the first subcutaneous immune globulin (IG) treatment approved for adult PI patients that allows a full monthly dose of IG to be administered with only one injection site and one infusion each month (once every three to four weeks depending on their established dosing regimen).
"With our swift commercial introduction, we look forward to sharing HYQVIA with adult PI patients seeking an option with less frequent dosing than current subcutaneous treatments without compromising efficacy, safety and tolerability. The goal of HYQVIA is to allow people with PI to feel less like patients by reducing the treatment burden associated with weekly dosing and multiple injection sites per dose," said Ludwig Hantson, Ph.D., President of Baxter BioScience.
HYQVIA was approved in Europe in 2013 for adults (≥18 years) with primary immunodeficiency syndromes and myeloma or chronic lymphocytic leukemia (CLL) with severe secondary hypogammaglobulinemia and recurrent infections. It is currently available in several European countries, including Germany, Netherlands, Sweden, Norway, Denmark, Ireland and Italy.
About HYQVIA
HYQVIA is an immune globulin with a recombinant human hyaluronidase indicated for the treatment of Primary Immunodeficiency (PI) in adults.
HYQVIA is a product consisting of Immune Globulin Infusion 10% (Human) (IG 10%) and recombinant human hyaluronidase (developed by Halozyme Therapeutics).
The IG component, a 10% solution that is prepared from large pools of human plasma consisting of at least 98% IgG, contains a broad spectrum of antibodies and provides the therapeutic effect. The Recombinant Human Hyaluronidase of HYQVIA increases dispersion and absorption of the Immune Globulin Infusion 10% (Human).