Table 1.

Description of studies and programs used for the safety summary.

Data Source (Sponsor/Marketing Authorization Holder)	Study or Program Description	Number of BAT Product 1-Treated Subjects/Patients	Status
BT-001 (EBCI) [23]	Phase 1, single-center, randomized, double-blind, parallel-arm safety and PK clinical study (U.S.). A single (1 vial) or double (2 vials) dose of BAT product was administered intravenously (IV) to healthy volunteers between 19 and 52 years of age. BAT product safety assessments were based on clinical observations, AEs, and laboratory tests 2 following administration.	N = 20 N = 20 (double dose)	Completed
BT-002 (EBCI) [22]	Phase 1b/2a, single-center, randomized, double-blind, parallel-arm, placebo-controlled safety and pharmacodynamic clinical study (U.S.) to evaluate the safety of BAT product and its effect in preventing paralysis induced by serotype A and serotype B botulinum toxins in the extensor digitorum brevis (foot) muscle. A single dose of BAT product was administered IV to healthy volunteers between 19 and 49 years of age. BAT product safety assessments were based on clinical observations, AEs, and laboratory tests 2 following administration.	N = 16	Completed
EAP (CDC) [24]	This expanded-access Investigational New Drug program (“compassionate use” IND) for the investigational BAT product implemented by the U.S. CDC collected safety and clinical benefit data prospectively and through medical record reviews of patients with confirmed or suspected botulism who were treated with investigational BAT product.	N = 249 (232 adults and 17 pediatric)	Completed
Post-marketing safety surveillance (EBCI)	Monitors the safety of BAT product post-licensure. Post-marketing AEs may be received from healthcare professionals, consumers, or from literature searches. Routine pharmacovigilance activities include case processing, benefit/risk assessment, and risk management plans.	N = 755	Ongoing
BT-010 (EBCI) [25]	A post-marketing BAT product registry to evaluate the safety and clinical outcomes of pediatric and adult patients following BAT product treatment for confirmed or suspected exposure to botulinum toxin and to estimate the absolute risk of hypersensitivity/allergic reactions, including serum sickness, febrile reactions, hemodynamic instability, bradycardia, and other SAEs in patients treated with BAT product.	N = 162(153 adults and 9 pediatric)	Completed

EBCI = Emergent BioSolutions Canada Inc.; EAP = expanded-access program; U.S. = United States of America; CDC = Centers for Disease Control and Prevention; AE = adverse event; SAE = serious adverse event. ¹ Each single-use vial of BAT product contains a minimum potency of 4500 Units (U) for serotype A antitoxin, 3300 U for serotype B antitoxin, 3000 U for serotype C antitoxin, 600 U for serotype D antitoxin, 5100 U for serotype E antitoxin, 3000 U for serotype F antitoxin, and 600 U for serotype G antitoxin. A single adult dose of BAT product is one vial, and pediatric dosing is a proportion of one vial based on body weight of the pediatric patient or 10% of adult dose regardless of body weight for infants. BAT is administered by slow intravenous (IV) infusion after dilution 1:10 in normal saline at the dose recommended in the product label. ² Laboratory tests include serum chemistry, hematology, urinalysis, 12-lead ECG, viral serology, pregnancy testing (serum and urine), as well as urine drug screening collected during the study.