Regulatory Decision Summary for Xembify

Primary Immunodeficiency (PI) diseases are a family of congenital disorders of the immune system that lead to an increase in frequency of infections, notably, but not limited to, bacterial infections of the respiratory tract.

To support safety and efficacy the Sponsor submitted a multi-center, prospective, open label, single-arm, single-sequence study conducted in the U.S. and Canada. The primary objective was to assess bioequivalence of Xembify (Immune Globulin Subcutaenous [IGSC], 20%), to Immune Globulin Intravenous [IGIV] Gamunex-C 10% (IGIV-C 10%) and to demonstrate the safety of Xembify. The study also provided sufficient data on the annualized rate of serious bacterial infections (SBI) to assess efficacy. The annualized SBI rate for Xembify (IGSC-20%) per subject year was 0.05. The most common adverse events in subjects receiving Xembify were infusion site reactions (ISR), including infusion site erythema, pain, swelling, bruising, nodule, pruritus, induration, scab and edema. Cough and diarrhea also occurred in >5% subjects during infusions of Xembify.

Immune globulins have been used to treat a variety of conditions and have been an established treatment for Primary Immunodeficiency since 1952. Immune globulins as a class have known risks of several adverse events, including infusion site reactions, hypersensitivity, aseptic meningitis, possible transmission of infectious diseases, renal dysfunction/failure, transfusion-related acute lung injury (TRALI) and risk of thrombosis and thromboembolic events. These risks are identified in the Xembify product monograph.

The nature and incidence of adverse events seen with Xembify are typical of products in this class of immune globulin subcutaneous (IGSC). Given the overall safety profile and absence of the occurrence of known risks for this class of products (such as thrombotic, hemolytic or other adverse events of special interest), the demonstration of bioequivalence to an approved immunoglobulin therapy for PI and documentation of a low rate of acute SBIs across seasons, a favorable benefit-risk profile has been determined.