The European Medicines Agency’s Committee for Medicinal Products for Human Use has adopted a positive opinion of fexinidazole, the first all-oral treatment that has been shown to be efficacious for both stages of sleeping sickness. This approval is a result of clinical trials led by the non-profit research and development organization DNDi and an application submitted by Sanofi. Swiss TPH contributed extensively to the development of this novel drug.
Sleeping sickness, or human African trypanosomiasis (HAT), is usually fatal without treatment. Transmitted by the bite of a tsetse fly, it causes neuropsychiatric symptoms; including aggression, psychosis and a debilitating disruption of sleep patterns that have given this neglected disease its name. About 65 million people in sub-Saharan Africa are at risk.
Fexinidazole is the first all-oral treatment that works both for the early stage as well as the second stage of the disease in which the parasites have crossed the blood-brain barrier, causing patients to suffer from neuropsychiatric symptoms. "This is an important decision which paves the way for the distribution of fexinidazole in endemic countries, and hence, opens a new era of treatment. Indeed, this first oral compound will become a key element in the elimination of this dreadful disease." said Christian Burri, Head of the Medicines Implementation Research Unit at Swiss TPH.
Important contribution to the development of fexinidazole
Swiss TPH has collaborated with DND i from the very beginning of the development of fexinidazole. The Parasite Chemotherapy Unit performed the assays necessary to identify fexinidazole as the most promising drug candidate against the Trypanosoma parasite. The Clinical Operations Unit provided scientific advice on the study protocols, assisted DND i in the management of the clinical trials in the Democratic Republic of the Congo (DRC) and was responsible for quality control. In Kinshasa, the capital of DRC, DND i and Swiss TPH share offices and collaborated very closely to implement the clinical trials in remote study centres.
"I had an opportunity to visit the field sites together with colleagues from DND i and Swiss TPH, which was an eye-opener in terms of barriers to access to health." said Jürg Utzinger, Director of Swiss TPH. "The partnership with DND i , Sanofi and various institutions in DRC is exemplary and demonstrates that - together - we are able to develop novel treatments that can change the life of the most neglected communities in our world."
Long-term engagement to fight sleeping sickness
Swiss TPH has been researching sleeping sickness ever since its foundation in 1943. Swiss TPH was the first institute to succeed in breeding tsetse flies under laboratory conditions. Subsequently, Swiss TPH developed culture systems for insects and pathogens, thereby laying the foundations for the compound screening laboratories. In the late 1990s, Swiss TPH with colleagues in DRC conducted the first modern clinical trial following international regulations on this disease.
At present, Swiss TPH is active in a wide range of research activities on sleeping sickness, from identifying new compounds and clinical research in low-resource settings to modelling various scenarios to halt the spread of the disease and pursue economic evaluations of different strategies for elimination of sleeping sickness.