Regulatory Decision Summary for BAT
Review decision
The Regulatory Decision Summary explains Health Canada’s decision for the product seeking market authorization. The Regulatory Decision Summary includes the purpose of the submission and the reason for the decision.
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What was the purpose of this submission?
The purpose of this submission is to obtain marketing authorization of BAT for the treatment of symptomatic botulism following documented or suspected exposure to botulinum neurotoxin serotypes A, B, C, D, E, F or G.
Human botulism is a serious, potentially fatal disease. However, it is relatively rare. Health Canada recognizes that there are circumstances in which sponsors cannot reasonably provide substantial evidence demonstrating the safety and efficacy of a therapeutic product in humans as there are logistical or ethical challenges in conducting the appropriate clinical trials. The Extraordinary Use New Drugs (EUND) pathway was used for authorization of BAT based on non-clinical and limited clinical information.
Why was the decision issued?
Human botulism is a serious, potentially fatal disease usually requiring intensive supportive care and mechanical ventilation. There is no authorized product to treat botulism in Canada.
As an EUNDS, BAT was assessed based on quality, non-clinical and limited clinical safety data. The efficacy of BAT was evaluated mostly based on the non-clinical data from the well-controlled, pivotal efficacy trials in symptomatic botulinum toxin challenged guinea pigs and rhesus macaques. These studies demonstrated that BAT significantly reduces the risk of death. Clinical data from an observational study in humans suggested the clinical benefits on the duration of hospitalization, ICU stay and mechanical ventilation uses in patients who are treated with BAT.
The reported adverse reactions were mostly mild or moderate. The serious adverse reactions reported were considered to be expected adverse reactions to equine hyper-immune globulin. The patients should be closely monitored during and following the administration of BAT (this information is presented in the product monograph).
Further safety and effectiveness data for BAT will be obtained in a post market study (patient registry). In addition, another post market study for a mass exposure scenario for the evaluation of safety and efficacy of BAT was planned in the United States. The study result would be submitted to Biologics and Genetic Therapies Directorate (BGTD) in case the event occurs in the United States.
The sale of BAT is restricted to Canadian federal, provincial/territorial and municipal governments as an authorized EUND.
Overall, the benefits outweigh the risks of BAT in the treatment of life-threating botulism patients. A Notice of Compliance (NOC) with post-decision commitments has been issued.
For more information on Health Canadas decision, please view the Summary Basis of Decision.
Decision issued
Approved; issued Notice of Compliance in accordance with the Food and Drug Regulations