Human African Trypanosomiasis (HAT)
Human African Trypanosomiasis (HAT) is a neglected tropical disease commonly known as African sleeping sickness. It is endemic to regions of sub-Saharan Africa and is a serious disease that is fatal if not adequately treated. HAT is caused by infection with protozoan parasites of the species Trypanosoma brucei and is vector-borne, i.e. transmitted through the bite of the tsetse fly.
The clinical course of HAT has two stages. In the first stage, the parasite is found in the peripheral circulation, but it has not yet infiltrated the central nervous system (“CNS”). Once the parasite crosses the blood-brain barrier and infects the CNS, the disease enters the second stage. The symptoms of the first stage include fever, headaches, pain in the joints and often irritation of the skin at the site of infection. The symptoms of the second stage infection include confusion and poor coordination, tremors, general motor weaknesses, irritability and aggressive behavior.
HAT is caused by infection with protozoan parasites of the species Trypanosoma brucei and is vector-borne, i.e. transmitted through the bite of the tsetse fly.
Treatment for HAT varies based on the form of the disease and the stage of the disease at diagnosis. Suramin has been in continuous use as the standard of care for the treatment of early stage East African sleeping sickness for more than 100 years. However, suramin has never been approved for use in the United States for any indication.
PaxMedica has exclusively licensed patient data from the East African hospitals where the disease occurs. We seek to leverage this patient data, in addition to its long history of use, in order to obtain an FDA approval for PAX-101. We believe that an approval of PAX-101 in HAT would qualify for New Chemical Entity exclusivity, Orphan Drug exclusivity, and could confer upon us the receipt of a Priority Review Voucher (PRV) by the FDA.