Takeda's HyQvia Approved for Humoral Immunodeficiency in Patients 2+

Takeda Canada Inc. has announced that Health Canada has extended the marketing authorization for HyQvia® for use in pediatric patients aged 2 years and older. HyQvia®, which is a subcutaneous immune globulin (SCIG) infusion, will serve as a replacement therapy for both primary humoral immunodeficiency (PI) and secondary humoral immunodeficiency (SI). Notably, HyQvia® is unique in that it can be administered every three or four weeks, making it a convenient treatment option for children and adolescents.

Whitney Ayoub Goulstone, Executive Director of ImmUnity Canada, highlighted the significance of this approval, stating, "This approval is significant as it gives children and adolescents living with immunodeficiencies another treatment option. Children living with immunodeficiencies can be severely impacted as it affects them physically, emotionally, and socially. Our organization welcomes treatment options for patients in Canada as this is to the benefit of better health outcomes and patient preference."

Primary immunodeficiencies (PI) encompass over 300 genetic defects and immune system disorders, varying widely in severity and symptoms. It is estimated that one in every 1,200 individuals is affected by PI, with early diagnosis and treatment being crucial for saving lives. In Canada, around 29,000 people suffer from PI, although more than 70% remain undiagnosed.

Jefferson Tea, Vice President of Medical & Scientific Affairs at Takeda, expressed enthusiasm for the new pediatric indication, stating, "The pediatric indication for HyQvia provides another treatment option for patients impacted by immunodeficiency disorders. We are committed to creating innovative solutions for Canadians living with immunodeficiency and are excited that parents and caregivers will have the option to support administration of this treatment to their children in their own home."

The authorization for use in pediatric patients is based on the results of two pivotal studies involving 66 patients aged between 2 and 16 years. The studies demonstrated that HyQvia was efficacious and exhibited no new safety concerns compared to its use in adults. In a Phase 3 clinical study involving 44 pediatric patients with PI, the treatment showed no clinically meaningful differences in trough IgG levels across different age groups. During the 12-month trial, HyQvia was effective in preventing acute serious bacterial infections (ASBIs), a primary endpoint of the study.

The mean rate of ASBIs per subject-year was 0.04, which was significantly lower than the threshold rate of 1.0 ASBI per subject-year, indicating the treatment's efficacy in pediatric subjects with PI. The overall rate of infections per subject-year was 3.20, with an upper limit of the 95% confidence interval at 4.05. Study 161503 also confirmed that the safety profile in children was similar to that in adults.

HyQvia® is designed as replacement therapy for both PI and SI in patients aged 2 years and older. This therapy is intended for individuals who lack sufficient antibodies or have weakened immune systems, leading them to experience frequent infections. The product monograph provides detailed information regarding contraindications, warnings, precautions, adverse reactions, interactions, dosing, and clinical use conditions.

Primary immunodeficiency disorders (PID) are a diverse group of conditions resulting from developmental defects and/or functional issues in the immune system. These disorders are broadly categorized into adaptive immunity disorders (such as T-cell, B-cell, or combined immunodeficiencies) or innate immunity disorders (such as phagocyte and complement disorders). Although clinical manifestations of PIDs can vary widely, they often involve increased susceptibility to infections.

Secondary immune deficiencies, or acquired deficiencies, are more common than primary immune deficiencies and arise from external factors such as viruses (e.g., HIV), severe malnutrition, chronic diseases (such as diabetes), immunosuppressive medication or chemotherapy, certain cancers (such as leukemia), and the absence of the spleen.