Immtech said that it plans to apply to the World Health Organization (WHO) to have pafuramidine designated as a WHO recommended drug, so that pafuramidine could be made more widely available.
The FDA also granted fast-track designation for this indication and, because of the favorable preclinical safety data for pafuramidine in reproductive and juvenile animals, the FDA allowed pregnant women and adolescents to be enrolled in this trial. Pregnant women, children and adolescents are known to be especially vulnerable to African sleeping sickness and to the risks of toxicity associated with current treatments.
Without effective treatment, African sleeping sickness is 100% fatal and has the propensity to develop into epidemics.
If approved, pafuramidine would become the first oral drug for African sleeping sickness. The availability of an easily administered oral therapy could help to expand treatment for first-stage African sleeping sickness, targeting the parasite as it circulates in the blood and before it infects the central nervous system.
The objective of this comparative trial is to assess the efficacy, safety and tolerability of pafuramidine versus pentamidine, the current standard therapy for African sleeping sickness. This interim analysis is expected to be completed in mid-year 2007.