European Medicines Agency Approves Label Update for HYQVIA® (Human Normal Immunoglobulin 10% and Recombinant Human Hyaluronidase), Expanding its Use to a Broader Group of Patients with Secondary Immunodeficiencies

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today announced that the European Medicines Agency (EMA) has approved a label update for HYQVIA^® (Human Normal Immunoglobulin 10% and Recombinant Human Hyaluronidase) broadening its use and making it the first and only facilitated subcutaneous immunoglobulin replacement therapy in adults, adolescents and children with an expanded range of secondary immunodeficiencies (SID). With the update, HYQVIA is now indicated for use as a replacement therapy in adults, children and adolescents (0-18 years) with SID who suffer from severe or recurrent infections, ineffective antimicrobial treatment and either proven scientific antibody failure (PSAF) or serum IgG level of <4 g/l.

The label update was based on the body of evidence from clinical trials that have demonstrated that subcutaneous administration of immunoglobulin has been shown to decrease the rate of infections in patients with SID and follows an update made by the EMA to the Summary of Product Characteristics (SmPC) for human normal immunoglobulin for intravenous administration (IVIg) which went into effect in 2019.^1,2 The efficacy and safety of HYQVIA has also been evaluated in a retrospective single-centre study in patients with immunodeficiencies secondary to haematologic malignancies.³ The safety profile of HYQVIA has remained unchanged with the expanded label.

“Over the past 10 years, we have seen demand for plasma-derived therapies, including Takeda immunoglobulin therapies, grow at an accelerated rate, reflecting how important these therapeutic options have become in the treatment of a variety of rare, life-threatening, chronic and genetic disease, including primary and secondary immunodeficiencies,” said Kristina Allikmets, Head of Plasma Derived Therapy Research & Development at Takeda. “With this expanded label, an expanded range of patients suffering from secondary immunodeficiencies now have HYQVIA as an option offering the flexibility of once-a-month treatment self-administered at home or with support of a medical professional in a healthcare setting.”

Takeda has a long-standing heritage in leading the development of innovative treatments for patients living with primary and secondary immunodeficiencies, which includes a broad immunoglobulin portfolio that can be tailored to each person’s unique treatment administration requirements.

About Immunodeficiency Disorders

Primary immunodeficiencies (PID) are a group of over 400 genetic disorders in which part of the body’s immune system is missing or does not function properly.⁴ Global estimates project that up to 6 million people may be living with PID, but only 650,000 people worldwide have been diagnosed.^5,6,7

Secondary immunodeficiency (SID) develops in patients who have a weakened immune system as a result of a disease or therapeutic intervention.⁸ Both conditions can result in impaired or insufficient antibodies, with risk of recurring serious infections and hospitalisation.⁸ SID has been estimated to be 30-fold more common than PID.⁹ The increased use of treatments that target B cells may also be contributing to the prevalence of SID.¹⁰ SID is often treated initially with prophylactic antibiotics. In patients where prophylactic antibiotics fail or are contraindicated, replacement immunoglobulin (IG) has been shown to effectively reduce infections.⁸

About HYQVIA^® (Human Normal Immunoglobulin 10% and Recombinant Human Hyaluronidase)

HYQVIA^® (Human Normal Immunoglobulin 10% and Recombinant Human Hyaluronidase) is a dual vial unit consisting of one vial of human normal immunoglobulin (IGI 10%) and one vial of recombinant human hyaluronidase (rHuPH20) for subcutaneous (SC) administration. The therapeutically active component of HYQVIA is human normal immunoglobulin (IGI 10%), manufactured from human plasma for fractionation compliant to European Pharmacopoeia. HYQVIA allows administration of IgG SC every 3 or 4 weeks as an alternative to IV administration or more frequent SC administration. In the EU, it is approved for use as a replacement therapy in adults, children and adolescents (0-18 years) in primary immunodeficiency syndromes with impaired antibody production and secondary immunodeficiencies in patients who suffer from severe or recurrent infections, ineffective antimicrobial treatment and either proven specific antibody failure (PSAF) or serum IgG level of <4 g/l.

HYQVIA Safety Information for Europe

Please consult the HYQVIA Summary of Product Characteristics (SmPC) before prescribing.

HYQVIA must not be used by patients with a hypersensitivity to human immunoglobulins, especially in very rare cases of IgA deficiency when the patient has antibodies against IgA. HYQVIA must not be used by patients with a systemic hypersensitivity to hyaluronidase or recombinant human hyaluronidase. HYQVIA must not be used by patients with a hypersensitivity to any of the excipients, including glycine.

HYQVIA must not be given intravenously.

Patients should be closely monitored and carefully observed for any adverse reactions throughout the infusion period, particularly patients starting with HYQVIA treatment. In case of adverse reaction, either the rate of administration must be reduced or the infusion stopped. The treatment required depends on the nature and severity of the adverse reaction.

In case of shock, standard medical treatment for shock should be implemented. Thromboembolic events (e.g. myocardial infarction, cerebral vascular accident, deep vein thrombosis, and pulmonary embolism), renal dysfunction/failure, aseptic meningitis syndrome, and hemolysis have been observed with IG 10% administered intravenously and cannot be excluded with use of HYQVIA. Thrombotic events and haemolysis have also been reported in association with the subcutaneous administration of immunoglobulin products.

Human normal immunoglobulin and human serum albumin (stabilizer of the recombinant human hyaluronidase) are produced from human plasma and may carry a risk of transmitting infectious agents.

About Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Diseases, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries.

For more information, visit https://www.takeda.com.

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¹ Cinetto F., Scarpa R., Neri R., Rizzo F., Compagno N. and Agostini C. (2017). "Subcutaneous Ig replacement therapy in primary and secondary hypogammaglobulinemia: A retrospective single centre study of 203 patients (Abst.)." Allergy European Journal of Allergy and Clinical Immunology Suppl. S103(72): 252-253.

² Vacca, A., Melaccio A., Sportelli A., Solimando A. G., Dammacco F. and Ria R. (2018). "Subcutaneous immunoglobulins in patients with multiple myeloma and secondary hypogammaglobulinemia: a randomized trial." Clin Immunol 191: 110-115.

³ Dimou, M., T. Iliakis, D. Maltezas, A. Bitsani, S. Kalyva, A. Koudouna, S. Kotsanti, P. Petsa, P. Papaioannou, M. C. Kyrtsonis and P. Panayiotidis (2018). "Efficacy-safety of Facilitated Subcutaneous Immunoglobulin in Immunodeficiency Due to Hematological Malignancies. A Single-Center Retrospective Analysis." Anticancer Res 38(7): 4187-4191.

⁴ Gruber C., Bogunovic D. Hum Genet. 2020 Feb 17. doi: 10.1007/s00439-020-02131-9.

⁵ Rezaei N et al. Introduction on Primary Immunodeficiency Diseases. In: Rezaei N, Aghamohammadi A, and Notarangelo LJ (Eds.), Primary Immunodeficiency Diseases 2017:1-81.

⁶ Parvaneh L. et al. Eur Ann Allergy Clin Immunol 2019; 51(1):32-37.

⁷ Lindegren M.L. et al. MMWR Recomm Rep 2004; 53:1-29.

⁸ Jolles S., Chapel H., & Litzman J. (2017). When to initiate immunoglobulin replacement therapy (IGRT) in antibody deficiency: a practical approach. Clinical and experimental immunology, 188(3), 333–341. https://doi.org/10.1111/cei.12915.

⁹ Monleón Bonet C et al., Expert Rev Clin Immunol. 2020

¹⁰ Patel S.Y., Carbone J. and Jolles S. (2019) The Expanding Field of Secondary Antibody Deficiency: Causes, Diagnosis, and Management. Front. Immunol. 10:33. doi: 10.3389/fimmu.2019.00033.

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