- Recommendation based on Phase II/III study demonstrating up to 96 per cent success rates at 18 months across both early and advanced stages of T.b. gambiense, the most common form of sleeping sickness
- The therapy, given as a single dose of three tablets, could offer a simpler alternative to longer, more complex regimens and help support the World Health Organization’s (WHO) goal of eliminating the disease by 2030
- Sanofi will donate the medicine to WHO through its philanthropic arm Foundation S
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has granted a positive opinion to Acoziborole Winthrop (acoziborole) as a single-dose oral treatment for both early- and advanced-stage gambiense sleeping sickness in adults as well as in adolescents 12 years and older weighing at least 40 kilograms.
A positive CHMP opinion through the EU-M4all procedure provides a strong endorsement that the medicine meets EU standards. This pathway, which engages WHO disease experts and regulators from endemic countries, is reserved for high-priority medicines addressing diseases with unmet medical needs. This decision will support regulatory approval of the treatment in the Democratic Republic of Congo (DRC) and lay the groundwork for a revision of the WHO’s sleeping sickness treatment guidelines. Such an update would ultimately broaden access to the medicine across other endemic countries in Central and West Africa.
Once approved in endemic countries, the medicine, co-developed by the Drugs for Neglected Diseases initiative (DNDi) and Sanofi, could provide a significant advance over current therapies. Existing treatments require either a 10-day course of oral medicine or a combination of injections and oral therapy for advanced cases.
Transmitted by the bite of an infected tsetse fly, human African trypanosomiasis, commonly known as sleeping sickness, is almost always fatal without treatment. In the early stage of the disease, people experience headaches or fever. In the late stage, the parasite crosses the blood-brain barrier and invades the central nervous system, causing behavioural, cognitive, and neurological symptoms, including seizures, sleep disturbance, aggression, confusion, lethargy, convulsions, and, ultimately, death.
DNDi conducted a pivotal Phase II/III study in the DRC and Guinea in partnership with national sleeping sickness control programmes, while Sanofi carried out the regulatory approval process. The CHMP positive opinion is based on clinical and non-clinical data provided by the partners, with efficacy and safety supported by the Phase II/III study, published in The Lancet Infectious Diseases medical journal, which demonstrated success rates at 18 months of up to 96 per cent across both stages of the disease with a good safety profile.
In 1998, nearly 40,000 cases of gambiense sleeping sickness were reported, with an estimated 300,000 undiagnosed. At the time, the only available treatment for those with the late stage of the disease was an injectable arsenic derivative with serious side effects. More than two decades of investment in new therapeutic tools resulted in increasingly improved treatments, including nifurtimox-eflornithine combination therapy in 2009 and the first oral treatment, fexinidazole, in 2018. In 2024, fewer than 600 cases of the disease were reported.
Sanofi will donate Acoziborole Winthrop to the WHO through its philanthropic organization, Foundation S – The Sanofi Collective. The medicine will be available free of charge to patients.
Another study underway in the DRC and Guinea is investigating Acoziborole Winthrop for the treatment of children ages 1 to 14.
