Botulism antitoxin heptavalent regulatory update

FDA's Blood Products Advisory Committee unanimously recommended approval of botulism antitoxin heptavalent (BAT) from Cangene to treat botulism. The committee said there is sufficient evidence from animal efficacy studies that BAT is "reasonably likely" to provide clinical benefit and that safety data from healthy volunteers support an acceptable risk/benefit profile. The panel also voted unanimously that animal studies and simulation modeling adequately support the proposed dosing regimen in adults, and voted 12-1, with 1 abstention, that the data support the proposed dosing in children. FDA said it will require a postmarketing study to monitor the safety of BAT. The agency said it would negotiate the surveillance period with the company after panel members expressed concerns that the three-year period initially proposed by the agency would not provide a sufficiently large data pool to make an accurate assessment of the product in pediatric patients.

Cangene is seeking approval for BAT under FDA's animal rule, which allows for marketing approval to be granted based on efficacy in relevant animal models and an acceptable safety risk profile in humans. The product is a heptavalent hyperimmune preparation containing neutralizing antibodies to the seven different botulinum toxin types. ...