AM25-ST-11-L - Saving Infant Lives Through Public–Private Partnership: The Development of an FDA-Approved Therapy for Infant Botulism

Infant botulism is a disease that mainly affects infants under one year of age. It was first recognized in 1976 by California Department of Public Health (CDPH) as a novel form of human botulism in which ingested spores of Clostridium botulinum colonize the infant's large intestine, producing botulinum neurotoxin in it, which causes weakness and flaccid paralysis. Infant botulism occurs globally and is the most common form of human botulism in the US.

Botulism Immune Globulin Intravenous [Human] (BIG-IV] is the only FDA approved treatment for infant botulism. To date, BIG-IV has been used to treat more than 3,000 infants worldwide and has resulted in more than 128 years of avoided hospital stay and more than $174 million of avoided hospital costs. BIG-IV consists of human-derived anti-botulinum toxin antibodies manufactured for CDPH from source plasma collected from a cohort of immunized donors in California and Ohio. To manufacture each lot, CDPH must partner with collection centers in these two regions. In 2024, CDPH required a new collection site in the Bay Area, and turned to Stanford Blood Center (SBC), a community blood center in Palo Alto. SBC diligently and rapidly built a source plasma program and successfully implemented source plasma collections for BIG-IV in 2024 and obtained source plasma licensure from the FDA.

The session will summarize the special circumstances that led CDPH to create, test, license, and distribute BIG-IV and describe how a community blood center implemented a licensed source plasma program specifically to support BIG-IV. Stories from families with infants treated with BIG-IV will also be shared.

Supported by an educational grant from Takeda Pharmaceuticals U.S.A., Inc.