OSAKA, Japan & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Takeda (TSE:4502/NYSE:TAK) today announced that the Japanese Ministry of Health, Labour and Welfare has approved the use of HYQVIA® [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] in patients with agammaglobulinemia or hypogammaglobulinemia1, disorders characterized by very low or absent levels of antibodies and an increased risk of serious recurring infection caused by primary immunodeficiency (PID) or secondary immunodeficiency (SID)2.

日本大阪和马萨诸塞州剑桥。-（商业新闻短讯）--武田（东京证交所：4502/纽约证交所：TAK）今天宣布，日本厚生劳动省已批准在患有无丙种球蛋白血症或低丙种球蛋白血症1的患者中使用HYQVIA®[免疫球蛋白输注10%（人类）和重组人透明质酸酶]，这些疾病的特征是抗体水平非常低或不存在，并且由原发性免疫缺陷（PID）或继发性免疫缺陷（SID）2引起的严重复发感染的风险增加。

The approval marks availability of the first and only facilitated subcutaneous immunoglobulin (fSCIG) therapy as a treatment option for appropriate patients in Japan..

该批准标志着日本第一个也是唯一一个促进皮下免疫球蛋白（fSCIG）治疗作为适当患者的治疗选择的可用性。。

HYQVIA is the first plasma-derived therapy for subcutaneous injection in Japan that consists of a combination of one vial of Immunoglobulin 10% and one vial of Recombinant Human Hyaluronidase PH20 (rHuPH20). The administration of rHuPH20 increases the dispersion and absorption of immunoglobulin (IG) in the subcutaneous tissue, allowing larger volumes to be infused in the infusion site.

HYQVIA是日本第一种用于皮下注射的血浆衍生疗法，由一瓶10%免疫球蛋白和一瓶重组人透明质酸酶PH20（rHuPH20）组成。rHuPH20的施用增加了免疫球蛋白（IG）在皮下组织中的分散和吸收，允许在输注部位输注更大体积。

This allows for less frequent dosing compared to other subcutaneous IG products, while avoiding the need for venous access. The ability to infuse a larger infusion volume is expected to increase administration flexibility for patients with agammaglobulinemia or hypogammaglobulinemia by decreasing the dosing frequency to once every 3 or 4 weeks, as compared to weekly or bi-weekly with conventional SCIG treatments..

与其他皮下IG产品相比，这可以减少给药频率，同时避免了静脉通路的需要。与常规SCIG治疗每周或每两周一次相比，通过将给药频率降低至每3或4周一次，预计输注更大输注量的能力将增加无丙种球蛋白血症或低丙种球蛋白血症患者的给药灵活性。。

The approval is based on data from two pivotal Phase 3, open-label, non-controlled studies evaluating the efficacy, safety, tolerability and pharmacokinetics in Japanese subjects with PID (NCT05150340, NCT05513586). In these studies, the efficacy and safety profile of HYQVIA in 16 patients aged 2 years or older in Japan were evaluated based on the results of the clinical trials.

该批准基于两项关键的第三阶段开放标签非对照研究的数据，这些研究评估了日本PID受试者的疗效，安全性，耐受性和药代动力学（NCT05150340，NCT05513586）。在这些研究中，根据临床试验的结果评估了HYQVIA在日本16名2岁或2岁以上患者中的疗效和安全性。

The Geo Mean of IgG trough level at the last 3 visits was 9.494g/L and was maintained at level comparable to treatment with intravenous or subcutaneous immunoglobulin (Geo Mean of IgG trough level 9.624g/L). The major adverse reactions were pyrexia 5 patients (31.3%) and infusion site erythema, injection site erythema, infusion site swelling, infusion site pain, and headache (12.5%)1.

最近3次就诊时IgG谷水平的地理平均值为9.494g/L，并维持在与静脉或皮下免疫球蛋白治疗相当的水平（IgG谷水平的地理平均值为9.624g/L）。主要不良反应为发热5例（31.3%），输液部位红斑、注射部位红斑、输液部位肿胀、输液部位疼痛、头痛（12.5%）1例。

Data from two Phase 3 clinical trials conducted in patients with PID in North America (NCT00814320, NCT01175213) was also included in the submission..

在北美对PID患者进行的两项3期临床试验（NCT00814320，NCT01175213）的数据也包括在提交的材料中。。

“We are delighted that HYQVIA, approved in more than 40 countries worldwide, has now been approved in Japan,” said Naoyoshi Hirota, Regional Head of Research & Development for Takeda’s Plasma-Derived Therapies Business Unit in Japan. “The subcutaneous IG therapies currently available in Japan for patients with agammaglobulinemia or hypogammaglobulinemia require infusion once every week or every 2 weeks.

武田血浆衍生疗法日本业务部门研发区域负责人Naoyoshi Hirota表示：“我们很高兴在全球40多个国家获得批准的HYQVIA现在已经在日本获得批准。”。“目前日本用于无丙种球蛋白血症或低丙种球蛋白血症患者的皮下IG疗法需要每周或每2周输注一次。

We are proud to offer Japanese patients the first and only facilitated subcutaneous treatment option that offers a reduced dosing frequency of every 3 or 4 weeks.”.

我们很自豪为日本患者提供第一个也是唯一一个方便的皮下治疗选择，每3或4周减少一次给药频率。”。

“There is a high unmet need for plasma-derived therapies (PDTs) in patients in Japan, which is anticipated to increase as education and timely diagnosis rates continue to improve,” said Kristina Allikmets, head of research & development for Takeda’s Plasma-Derived Therapies Business Unit. “The approval of HYQVIA, the first and only facilitated SCIG treatment, is further evidence of Takeda’s commitment to add to the standard of care for patients in Japan.

武田血浆衍生疗法业务部门研发主管克里斯蒂娜·阿利克梅茨（KristinaAllikmets）表示：“日本患者对血浆衍生疗法（PDTs）的需求很高，预计随着教育程度和及时诊断率的不断提高，需求会增加。”。“第一个也是唯一一个促进SCIG治疗的HYQVIA的批准，进一步证明了武田致力于提高日本患者的护理标准。

We look forward to continuing to bring new therapeutic options that support and enhance the experience of patients in our home country throughout the next decade.”.

我们期待着在未来十年内继续带来新的治疗选择，以支持和提高我们祖国患者的体验。”。

With this approval, Takeda is now able to offer a range of SCIG therapies to patients based on their individual administrative needs, reflecting the company’s commitment to offer patients in Japan a broader choice of treatment options. It also follows the announcement of a significant investment to build a new manufacturing facility for plasma-derived therapies (PDTs) in Osaka, Japan.

有了这一批准，武田现在能够根据患者的个人管理需求为患者提供一系列SCIG治疗，这反映了该公司致力于为日本患者提供更广泛的治疗选择。此前，日本宣布将在大阪投资兴建一座新的血浆衍生疗法（PDT）生产设施。

HYQVIA is also currently under review in Japan for additional indications..

HYQVIA目前也正在日本审查其他适应症。。

About Agammaglobulinemia or Hypogammaglobulinemia

关于无丙种球蛋白血症或低丙种球蛋白血症

Agammaglobulinemia is an inherited disorder caused by a gene defect that blocks the growth of normal, mature immune cells called B lymphocytes3. Hypogammaglobulinemia is a condition in which patients have low levels of antibodies due either to inherited genetic conditions (PID) or secondary effects (SID) – like chemotherapy, certain comorbid disorders, or immunosuppressants2.

无丙种球蛋白血症是一种遗传性疾病，由基因缺陷引起，该基因缺陷会阻止称为B淋巴细胞的正常成熟免疫细胞的生长3。低丙种球蛋白血症是一种由于遗传性遗传病（PID）或继发性效应（SID）如化疗，某些合并症或免疫抑制剂而导致患者抗体水平低的疾病2。

Individuals with any form of antibody deficiency frequently experience recurring and/or severe infections, and immunoglobulin replacement therapy can increase the level of antibodies in the body..

患有任何形式抗体缺乏症的个体经常会复发和/或严重感染，免疫球蛋白替代疗法可以增加体内抗体的水平。。

About Primary Immunodeficiency and Secondary Immunodeficiency

关于原发性免疫缺陷和继发性免疫缺陷

Primary immunodeficiency describes a heterogeneous group of more than 480 rare genetic diseases wherein part of the immune system is missing or not functioning properly4. Secondary immunodeficiency is defined as an acquired impairment of the immune response resulting from an underlying condition or factors extrinsic to the immune system.

原发性免疫缺陷描述了480多种罕见遗传疾病的异质性群体，其中部分免疫系统缺失或功能不正常4。继发性免疫缺陷被定义为由免疫系统外在的潜在病症或因素引起的免疫应答的获得性损伤。

SID may occur as a consequence of malnutrition, metabolic disorders, use of immunosuppressive medications, chronic infections, malignancies, or severe trauma5. Due to their impaired immune system, patients with PID and SID may be more susceptible to infection, and it may take longer to recover from it.

SID可能是营养不良，代谢紊乱，使用免疫抑制药物，慢性感染，恶性肿瘤或严重创伤的结果5。由于免疫系统受损，PID和SID患者可能更容易感染，并且可能需要更长的时间才能恢复。

In patients with antibody deficiency and increased susceptibility and/or persistent infections, substitution with functional antibodies (immunoglobulin replacement therapy) is the standard of care to support the immune system's functioning5..

对于抗体缺乏和易感性增加和/或持续感染的患者，用功能性抗体替代（免疫球蛋白替代疗法）是支持免疫系统功能的标准护理5。。

About HYQVIA®

关于HYQVIA®

HYQVIA is the combination product for subcutaneous injection containing one each vial of Subcutaneous Immunoglobulin 10% (SCIG10%) and Recombinant Human Hyaluronidase PH20 (rHuPH20). As of May 2024, HYQVIA has been approved in more than 40 countries worldwide.

HYQVIA是用于皮下注射的组合产品，每瓶含有一种皮下免疫球蛋白10%（SCIG10%）和重组人透明质酸酶PH20（rHuPH20）。截至2024年5月，HYQVIA已在全球40多个国家获得批准。

HYQVIA Product Overview in Japan

HYQVIA在日本的产品概述

Brand Name

品牌名称

HYQVIA 10% Subcutaneous Injection Set 5g/50mL 10g/100mL 20g/200mL

HYQVIA 10%皮下注射组5g/50mL 10g/100mL 20g/200mL

Generic Name

通用名称

pH4 Treated Acid Human Globulin (Subcutaneous Injection)

pH4处理的酸性人球蛋白（皮下注射）

Vorhyaluronidase Alfa (Genetical Recombination)

伏透明质酸酶α（基因重组）

Indications

适应症

Agammaglobulinemia or Hypogammaglobulinemia

无丙种球蛋白血症或低丙种球蛋白血症

Dosage and Administration

剂量和给药

Human immunoglobulin G is administered subcutaneously at the same site within approximately 10 minutes after the subcutaneous injection of vorhyaluronidase alfa (genetical recombination).

在皮下注射伏透明质酸酶α（基因重组）后约10分钟内，在同一部位皮下注射人免疫球蛋白G。

Initiate the treatment with human immunoglobulin G and vorhyaluronidase alfa (genetical recombination) at 1/3 or 1/4 of the doses described below, and then the doses are titrated up. Note that the dosing interval should be extended depending on the doses.

以下述剂量的1/3或1/4开始用人免疫球蛋白G和伏透明质酸酶α（基因重组）治疗，然后滴定剂量。注意，应根据剂量延长给药间隔。

•Usually, 150 – 600 mg (1.5 – 6 mL)/kg of body weight of human immunoglobulin G is administered once every 3 weeks or 200 – 800 mg (2 – 8 mL)/kg of body weight of human immunoglobulin G is administered once every 4 weeks.

•通常，每3周服用150–600 mg（1.5–6 mL）/kg体重的人免疫球蛋白G，或每4周服用200–800 mg（2–8 mL）/kg体重的人免疫球蛋白G。

•Vorhyaluronidase alfa (genetical recombination) is administered at the dose of 80 units (0.5 mL) per 1 g of human immunoglobulin G.

•伏透明质酸酶α（基因重组）的剂量为每1克人免疫球蛋白g 80单位（0.5毫升）。

The dose and the number of doses per 3 or 4 weeks may be adjusted according to the patient’s condition.

每3周或4周的剂量和剂量数量可根据患者的病情进行调整。

About Takeda

关于武田

Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines.

武田致力于为人们创造更好的健康，为世界创造更美好的未来。我们的目标是在我们的核心治疗和业务领域发现并提供改变生命的治疗方法，包括胃肠道和炎症、罕见疾病、血浆衍生疗法、肿瘤学、神经科学和疫苗。

Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet.

与我们的合作伙伴一起，我们的目标是通过我们动态多样的渠道改善患者体验，并推进治疗选择的新前沿。作为总部位于日本的领先的基于价值观、研发驱动的生物制药公司，我们以对患者、人民和地球的承诺为指导。

Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit www.takeda.com..

我们在大约80个国家和地区的员工是由我们的目标驱动的，并以两个多世纪以来定义我们的价值观为基础。有关更多信息，请访问www.takeda.com。。