CBER: Guidance for Industry: Safety, Efficacy, and Pharmacokinetic Studies to Support Marketing of Immune Globulin Intravenous (Human) as Replacement Therapy for Primary Humoral Immunodeficiency

We, FDA, are providing you, Investigational New Drug Application (IND) sponsors and Biologics License Application (BLA) applicants, recommendations for testing the safety and efficacy of Immune Globulin Intravenous (Human)(IGIV) products as replacement therapy in primary humoral immunodeficiency. The document provides guidance on general principles concerning clinical trial design to evaluate safety, efficacy, and pharmacokinetics of investigational IGIV products and is intended to assist you in the preparation of the clinical/biostatistical and human pharmacokinetic sections of a BLA. This guidance does not address evidence of clinical efficacy for other indications, or other sections of a BLA such as chemistry, manufacturing, and controls and preclinical toxicology.