European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion of Fexinidazole Winthrop (fexinidazole) for both stages of sleeping sickness.
The approval comes after clinical trials led by the non-profit research and development organization Drugs for Neglected Diseases Initiative (DNDi), and the regulatory submission by Sanofi.
The drug, a 5-nitroimidazole derivative, was rediscovered in 2005 through a collaboration with the Swiss Tropical Public Health Institute, during DNDi’s search for anti-parasitic compounds. It was originally discovered by Hoescht, now known as Sanofi, in the 1980s but was abandoned for “strategic reasons.”
Since its rediscovery, Sanofi will be responsible for industrial development, registration, production, and distribution, with DNDi managing preclinical, clinical, and pharmaceutical development.
Bernard Pecoul, DNDi executive director, said in a statement that the drug is, “an entirely new chemical entity that has been developed through an alternative non-profit R&D Model.” It is also the first new chemical entity to be developed by DNDi.
In 2017, Sanofi submitted a regulatory dossier to the EMA. To develop fexinidazole, DNDi spent €55m ($62.5m), including preclinical development and clinical studies. The project was supported by seven European countries and private donors, including the Bill & Melinda Gates Foundation and Médecins Sans Frontières.
Fexinidazole and sleeping sickness
Fexinidazole is the first all-oral treatment shown to be efficacious for both stages of sleeping sickness. It is indicated for a once-a-day, 10-day course of treatment. Clinical trials enrolled 749 patients in the Democratic Republic of Congo (DRC) and Central African Republic. The trials demonstrated high efficacy and safety in both adults and children.
The previous treatment for sleeping sickness was an “arsenic derivative that killed 5% of all patients,” according to Victor Kande, neglected tropical diseases expert advisor to the Ministry of Health in the DRC. He further explained, that current treatments “require a patient to be hospitalized and pose a huge logistical burden on the health system. Fexinidazole comes as a simple pill: this is a huge leap in how we can tackle this deadly disease.”
Sleeping sickness is usually fatal without treatment. The disease is transmitted through the tsetse fly and causes neuropsychiatric symptoms and debilitating disruption to sleep patterns. It largely affects the people in sub-Saharan Africa.
Fexinidazole works for both stages of the disease, the early stage and the second stage, in which the parasites have crossed the blood-brain barrier and caused neuropsychiatric symptoms to arise.