The US FDA has approved fexinidazole as the first all-oral treatment for both stages of the Trypanosoma brucei gambiense form of sleeping sickness (Human African trypanosomiasis) in patients above 6 years of age and weighing at least 20 kg.
Fexinidazole was developed as part of an innovative partnership between the non-profit research and development organization Drugs for Neglected Diseases initiative (DNDi) and Sanofi for the treatment of Trypanosoma brucei gambiense human African trypanosomiasis (HAT), more commonly known as sleeping sickness. In November 2018, based on the clinical trial results, the drug received a positive scientific opinion from the European Medicines Agency (EMA) followed by a marketing authorization in Democratic Republic of Congo (DRC) in January 2019.
Fexinidazole is the first New Chemical Entity (NCE) developed by DNDi, which has steered its progression through all stages of the drug development pipeline from lab to patient. DNDi initiated the development of Fexinidazole in 2005 in collaboration with Swiss Tropical and Public Health Institute. Upon completion of pre-clinical studies in 2009, DNDi partnered with Sanofi for further development, manufacturing, and distribution of fexinidazole.
Fexinidazole is indicated as a 10-day once-a-day treatment, offering significant advantages over the previous standard of care, NECT, because it eliminates the need for systematic hospitalization and leads to a reduction in the number of lumbar punctures. Fexinidazole is the first all-oral treatment that works at both first and second stage of the disease in which the parasites have crossed the blood-brain barrier, causing patients to suffer from neuropsychiatric symptoms.
Sanofi has donated Fexinidazole free of cost to WHO for distribution to national sleeping sickness control programmes in disease-endemic countries. Distribution in the Democratic Republic of Congo began in January 2020. As a result of FDA approval, a Tropical Disease Priority Review Voucher (PRV) has been awarded to DNDi. The FDA Tropical Disease PRV Program was established in 2007 to incentivize development of new treatments for neglected tropical diseases, including sleeping sickness and any benefits from the PRV will be shared between Sanofi and DNDi. These programs incentivize large pharma companies in to support development of treatments for neglected disease and enhance partnerships at early stages of development.