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历经2年磨难,FDA终于批准百特皮下注射药物HyQvia

来源:生物谷 2014-09-15 10:22

2014年9月15日讯 /生物谷BIOON/ --历经2年磨难,百特和合作伙伴Halozyme皮下注射药物HyQvia终于获FDA批准,用于原发性免疫缺陷症(PI)的治疗。2年前,FDA因安全性问题拒绝批准HyQvia。

百特(Baxter)9月12日宣布,FDA已批准皮下注射(SC)药物HyQvia,用于原发性免疫缺陷症(PI)成人患者的治疗。今年7月底,FDA血液制品专家委员会(BPAC)以15比1的投票结果,认为HyQvia用于治疗原发性免疫缺陷症(PI)具有良好的风险/利益属性,建议批准HyQvia用于PI的治疗。百特预计在未来几周内将HyQvia推向市场。

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IGSC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提供全部的治疗剂量。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。

2012年8月,FDA因安全性问题拒绝批准HyQvia,百特和合作伙伴Halozyme称,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),以便重新启动关于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单一的皮下注射位点注入一剂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。(生物谷Bioon.com)

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英文原文:FDA Approves Baxter's HYQVIA for Treatment of Adults with Primary Immunodeficiency 

DEERFIELD, Ill. and SAN DIEGO, Calif., September 12, 2014 - Baxter International Inc. (NYSE:BAX) and Halozyme Therapeutics, Inc., (NASDAQ:HALO) today announced that the United States Food and Drug Administration (FDA) approved Baxter's subcutaneous treatment for adult patients with primary immunodeficiency (PI), HYQVIA [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase].

HYQVIA is the first subcutaneous immune globulin (IG) treatment approved for PI patients with a dosing regimen requiring only one infusion up to once per month (every three to four weeks) and one injection site per infusion to deliver a full therapeutic dose of IG. The majority of PI patients today receive intravenous infusions in a doctor's office or infusion center, and current subcutaneous IG treatments require weekly or bi-weekly treatment with multiple infusion sites per treatment.

"Patients with PI value treatments that offer efficacy, safety and tolerability. Since each person with PI responds differently to treatment, having options that meet these individual needs is critically important," commented Marcia Boyle, President and Founder of the Immune Deficiency Foundation. "We commend Baxter for its significant commitment and investment in the development of HYQVIA."

"The availability of HYQVIA has a significant impact on the treatment of PI, allowing for effective delivery of a full therapeutic dose of IG less frequently than other subcutaneous treatments (up to once a month), while maintaining the efficacy, safety and tolerability profile that is most important for patients," said Ludwig Hantson, Ph.D., President of Baxter BioScience. "This approval highlights the support of the patient community for new treatment options."

"Today's FDA approval of HYQVIA is a significant milestone for Halozyme as it represents the first U.S. approved Biologics License Application which utilizes our rHuPH20 platform," commented Dr. Helen Torley, President and Chief Executive Officer of Halozyme. "I would like to thank the talented teams at both Halozyme and Baxter for their dedication to bring a new treatment alternative to PI patients managing a life-long disease."

Baxter expects to launch HYQVIA in the U.S. in the coming weeks. 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 being prescribed in several European countries, including Germany, Netherlands, Sweden, Norway, Denmark, Ireland and Italy.

About HYQVIA
HYQVIA is an immune globulin with a recombinant human hyaluraonidase 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).

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