Sanofi, in collaboration with DNDi and the HAT-r-ACC consortium, announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive scientific opinion for Fexinidazole Winthrop. This marks the first oral treatment for the acute form of sleeping sickness (rhodesiense) in both adults and children aged six and older, weighing at least 20 kg. The approval covers both first-stage (haemo-lymphatic) and second-stage (meningo-encephalitic) Trypanosoma brucei (T.b.) rhodesiense sleeping sickness, a severe and lethal form prevalent in Eastern and Southern Africa.
This CHMP opinion follows Sanofi's application under Article 58 and clinical trials conducted in Malawi and Uganda by the non-profit medical research organization Drugs for Neglected Diseases initiative (DNDi). In 2018, CHMP issued a positive opinion for Fexinidazole Winthrop as the first all-oral treatment for the more common T.b. gambiense form of sleeping sickness found in West and Central Africa.
Sleeping sickness, or human African trypanosomiasis (HAT), is a potentially fatal disease transmitted by infected tsetse flies found in 36 African countries. It manifests with neuropsychiatric symptoms, disrupting sleep patterns and leading to death.
For the T.b. rhodesiense variant, Fexinidazole Winthrop is indicated as a 10-day, once-daily oral treatment. Recent data from DNDi’s Phase 2/3 clinical trial, presented at the European Congress of Tropical Medicine and International Health, demonstrated the high effectiveness and safety of Fexinidazole Winthrop in treating T.b. rhodesiense sleeping sickness. In 12 months of post-treatment follow-up evaluations, only one patient (2.94%) with the advanced form of the disease relapsed, requiring treatment with the standard arsenic derivative.
While T.b. gambiense primarily infects humans, T.b. rhodesiense is a zoonotic disease, spreading from animals to humans. Common reservoirs include cattle and wild animals like bushbucks and zebras. The CHMP opinion facilitates the update of WHO guidelines on sleeping sickness treatment, and the distribution of Fexinidazole Winthrop by WHO in African countries where T.b. rhodesiense is prevalent. Sanofi’s philanthropic organization, Foundation S, will donate Fexinidazole Winthrop to WHO.
Already registered in the Democratic Republic of the Congo and Uganda for T.b. gambiense treatment, Fexinidazole Winthrop is recommended for use in an additional 10 African countries, including Angola, Burkina Faso, Central African Republic, Chad, Congo, Côte d'Ivoire, Equatorial Guinea, Gabon, Guinea, and South Sudan.