The registration follows successful clinical trials conducted in the DRC and Guinea and paves the way for use across all African countries where the most common form of the disease, caused bygambiensehuman African trypanosomiasis, is endemic. The medicinewill be available free of charge to patients.
The Democratic Republic of the Congo (DRC) has approved the use of Acoziborole Winthrop (acoziborole) as a single-dose oral treatment for both early- and advanced-stage gambiense sleeping sickness in adults and adolescents 12 years and older weighing at least 40 kilograms.
The decision follows Phase II/III clinical trials conducted in the DRC and in Guinea by the medical research non-profit organization Drugs for Neglected Diseases initiative (DNDi), in partnership with the DRC and Guinea’s national sleeping sickness control programmes. The studies demonstrated that acoziborole is safe and has a cure rate of up to 96 per cent across both stages of the disease.
Acoziborole was co-developed by DNDi and the pharmaceutical company Sanofi. It will be available free of charge to patients.
In 2024, the DRC reported 330 cases of sleeping sickness, accounting for more than half of all cases reported across Africa that year—a steep decline from the late 1990s. In 1998, nearly 40,000 cases of gambiense sleeping sickness were reported on the continent, with an estimated 300,000 undiagnosed.
The DRC has been at the forefront of clinical research and registration of several new treatments against the disease. The latest, acoziborole, will be a crucial tool in reaching the final mile to sustainably eliminate a disease that has killed millions of Africans over the past century.
‘The registration of acoziborole in the DRC is fantastic news for patients. The drug represents a major advance over existing treatments: it does not require hospitalization, is well tolerated, and can be administered immediately after diagnosis. This will allow us to reach the most remote endemic areas. With this new tool, we can realistically aim at eliminating the disease in the coming years,’ said Dr Erick Miaka, Director of the DRC’s national sleeping sickness control programme.
Existing treatments require either a 10-day course of oral medicine or, for patients in the most advanced stage of the disease, a combination of injection and oral therapy that requires several days of hospitalization.
In February 2026, acoziborole received a positive opinion from the European Medicines Agency through the EU-M4all procedure, providing a strong endorsement that the medicine meets high quality standards. Following registration in the DRC, the approval is expected to enable other sleeping sickness-endemic countries in Central and West Africa, including Guinea, to authorize importation and accelerate access to the new medicine.
‘Guinea succeeded in eliminating sleeping sickness as a public health problem last year—a remarkable achievement driven by research, science, and human perseverance. But the disease can return if we become complacent. Acoziborole, a breakthrough treatment successfully tested here in Guinea, will be a powerful tool to prevent any resurgence, as it is safe, exceptionally simple to administer, and can be taken in just one day,’ said Mamadou Camara, Guinea’s Coordinator of the National Human African Trypanosomiasis (the scientific name of the disease) Control Programme.
‘With foreign aid and global health in crisis, the development of acoziborole represents a resounding success for African science and global cooperation. It is powerful proof that alternative models of medical research, based on patients’ needs, can deliver new breakthrough medicines. We are delighted that the communities most affected by the disease will finally have access to this treatment they played a major role in developing,’ said Chirac Bulanga, West and Central Africa Director at DNDi.
‘As mistrust of health workers fans the flames of the current Ebola epidemic in Ituri, our experience with sleeping sickness shows that long-term investment in research and development can regain the trust of communities. The same communities that suffered under arsenic-based treatments two decades ago are now partners with our health workers, thanks to innovative new treatments like acoziborole,’ Bulanga added.
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.
Until two decades ago, the only available treatment for patients with the late stage of the disease was an injectable arsenic derivative with serious side effects. More than 20 years 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.
Sanofi, the market-authorization holder, will donate acoziborole to the World Health Organization through its philanthropic organization, Foundation S – The Sanofi Collective.
Acoziborole has also been evaluated in children aged 1 to 14 years in a clinical trial conducted in the DRC and Guinea, generating data to support its use in children, who have historically faced even greater neglect than adults when it comes to treatment.
About DNDi’s sleeping sickness programme
Acoziborole Winthrop is the first oral single-dose new chemical entity to be issued from DNDi’s lead optimization programme for sleeping sickness. It started with an initial hit identified in the chemical library of Anacor Pharmaceuticals, which was acquired by Pfizer in 2016. The initial structure was then optimized with Scynexis and Pace University and was then selected as a candidate for development and Phase I safety studies conducted successfully in France, the UK, and Malaysia.
Acoziborole Winthrop is the latest innovation, the result of more than two decades of scientific collaboration involving DNDi, Sanofi, and partners. In 2009, they developed a combination of existing drugs known as NECT, which was extremely effective and had a good safety profile. A donation programme was set up by Sanofi and Bayer to provide NECT free of charge to endemic countries through the World Health Organization (WHO), radically improving treatment options for patients.
About DNDi
The Drugs for Neglected Diseases initiative (DNDi) is a not-for-profit medical research organization that discovers, develops, and delivers safe, effective, and affordable treatments for neglected populations. DNDi is developing medicines for sleeping sickness, leishmaniasis, Chagas disease, parasitic worm diseases, mycetoma, dengue, paediatric HIV, cryptococcal meningitis, and hepatitis C. Its research priorities include children’s health; gender equity and gender-responsive R&D; and diseases impacted by climate change. Since its creation in 2003, DNDi has collaborated with public and private partners worldwide to deliver new treatments for six deadly diseases, saving millions of lives. Acoziborole is the 14th treatment delivered by DNDi. dndi.org
DNDi media relations
Frédéric Ojardias – Geneva | +41 79 431 62 16 | fojardias@dndi.org
Linet Atieno Otieno – Nairobi | +254 733 624 206 | latieno@dndi.org
Ilan Moss – New York| +1 646 266 5216 | imoss@dndi.org
Photo credit: Brent Stirton/Getty Images for DNDi
