Advance the clinical development of oxfendazole as an anti-parasitic macrofilaricidal treatment
current phase of drug development
updated 16 Feb 2023
Screening of drug libraries obtained from biotech and pharmaceutical companies, along with active screening of the literature, identified oxfendazole, a veterinary product used for deworming in animals, as a potential macrofilaricidal treatment for river blindness.
Based on highly encouraging pre-clinical data on the efficacy of oxfendazole, DNDi is evaluating the use of oxfendazole for filarial infections. DNDi is moving ahead with pharmaceutical development of the drug, and the HELP Consortium, of which DNDi is a member, is conducting a Phase I clinical trial.
HELP Consortium: an international network to speed up identification of new treatments for worm-related diseases
Launched in 2019, the Helminth Elimination Platform (HELP) led by the Swiss Tropical and Public Health Institute (Swiss TPH) convenes key players in research and development for filarial diseases. The multidisciplinary consortium works to identify new treatments against nematode worms, including onchocerciasis, lymphatic filariasis, hookworm, and whipworm.
All regulatory approvals were obtained for the Phase I study in Tanzania, sponsored by Swiss Tropical and Public Health Institute (Swiss TPH) and conducted by the Ifakara Health Institute in Tanzania. The Phase I clinical trial assessing the bioavailability of oxfendazole was initiated and final follow-up visits were completed for the first two patient cohorts. Two safety review committee meetings were held, and no safety concerns were noted.
In 2021, we began preparing for a Phase I trial to test the bioavailability of an oxfendazole tablet that is field adapted and easy to use. The trial will be sponsored by the Swiss Tropical and Public Health Institute and conducted by the Ifakara Health Institute in Tanzania.
No serious adverse events occurred in the single ascending dose or multiple ascending dose studies (NCT02234570 and NCT03035760, clinicaltrial.gov) conducted by the Oxfendazole Development Group under the auspices of the US National Institute of Allergy and Infectious Diseases. Based on the data in healthy volunteers, oxfendazole applied orally in a liquid formulation was safe and well tolerated at doses up to 60 mg/kg and showed acceptable safety and tolerability profiles after five repeated daily doses of up to 15 mg/kg, with a few reports of non-serious adverse events in the two studies. There was no increase in adverse events with an increased dose.
Therefore, the available data suggest that oxfendazole is safe for use in humans and a Phase I bioavailability study is planned using a solid, field-adapted tablet. With recently obtained funding from the European Union’s Horizon 2020 research and innovation programme, the HELP Consortium will conduct a bioavailability Phase I trial in Tanzania to evaluate the safety, tolerability, and pharmacokinetics of the tablet.
DNDi developed a field-adapted tablet.
News & resources
- 7 April 2023 - eWHORM project launched to eliminate worm infections in Sub Saharan Africa
- 19 October 2022 - Comparison of the macrofilaricidal efficacy of oxfendazole and its isomers against the rodent filaria Litomosoides sigmodontis, Frontiers in Tropical Diseases
- 6 July 2020 - Oxfendazole mediates macrofilaricidal efficacy against the filarial nematode Litomosoides sigmodontis in vivo and inhibits Onchocerca spec. motility in vitro, PLOS Neglected Tropical Diseases
Get our latest news, personal stories, research articles, and job opportunities.