FDA extends approval of Gammaplex (immune globulin intravenous) to pediatric patients with primary immunodeficiency- Bio Products Lab

Bio Products Lab announced the FDA approval of Gammaplex (immune globulin intravenous [human], 5% liquid) for pediatric patients two years of age and older who have primary humoral immunodeficiencies. The approval was based on data submitted to the FDA as part of a post-marketing commitment following approval of Gammaplex for replacement therapy in adults in 2009.

Data supporting the approval was generated from a clinical study involving 25 children and adolescents with primary immunodeficiencies aged 3-16 years who were treated with Gammaplex for 12 months. The study’s primary efficacy endpoint was the incidence of serious, acute bacterial infections (SABIs) as defined by the FDA. Secondary objectives assessed safety and tolerability. Throughout the course of the study, two SABIs of pneumonia were reported, resulting in an annual SABI event rate of 0.09 (upper, 1-sided 99% CI, 0.36), well below the maximum SABI event rate of 0.5 per subject required for approval.

Fourteen subjects (56%) had an adverse reaction at some time during the clinical trial that was considered product-related. Of these 14 subjects, two had adverse reactions that were considered definitely related to Gammaplex including headache, fatigue, and myalgia. The most common adverse reactions, occurring in ≥5% of subjects were dyspnea (2/25 subjects, 8%), otitis media acute (2/25 subjects, 8%), and tonsillar disorder (2/25 subjects, 8%). Two subjects reported a serious adverse event of lobar pneumonia. Neither serious adverse reaction was considered related to Gammaplex, and neither met FDA-defined SABI criteria. No subjects withdrew from the study due to adverse events.