Michael Gwarisa
A breakthrough in the fight against sleeping sickness could transform care for thousands of patients in Africa. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has given a positive opinion to Acoziborole Winthrop, a single-dose, three-tablet treatment for the most common form of the disease, gambiense sleeping sickness. Developed by the Drugs for Neglected Diseases initiative (DNDi) and Sanofi, the therapy is effective for both early and advanced stages in adults and adolescents aged 12 and older weighing at least 40 kilograms.
Sleeping sickness, or human African trypanosomiasis, is transmitted by the bite of infected tsetse flies and is almost always fatal if untreated. Early-stage symptoms include headaches and fever, but as the disease progresses, the parasite invades the central nervous system, causing severe neurological and behavioural disturbances, including sleep disruption, confusion, seizures, and eventually death. Historically, treatments were complex and risky, often involving injections or arsenic derivatives with severe side effects.
The new single-dose therapy represents a dramatic simplification of care. Phase II/III clinical trials conducted in the Democratic Republic of Congo (DRC) and Guinea showed success rates of up to 96 percent at 18 months across both early and late-stage patients. The trials, run in partnership with national sleeping sickness control programs, demonstrated the safety and efficacy of the pill while highlighting the power of Africa-led research.
Dr. Luis Pizarro, Executive Director of DNDi, called the development “a transformative step toward finally eliminating sleeping sickness, a disease that has devastated communities across Africa for over a century.”
He added that the single-day treatment could significantly reduce the logistical burden on health systems and improve patient adherence compared to the longer, multi-day regimens previously used.
Sanofi will donate the medicine through its philanthropic arm, Foundation S – The Sanofi Collective, making it freely available to patients in endemic countries.
Audrey Duval, Sanofi’s Executive Vice President for Corporate Affairs, emphasized the company’s ongoing commitment to tackling neglected diseases in partnership with WHO, DNDi, and other stakeholders. “Acoziborole builds on decades of innovation and collaboration, helping drive reported cases to historic lows and moving us closer to the WHO’s 2030 elimination goals,” she said.
The CHMP positive opinion was granted through the EU-M4all procedure, a special pathway for medicines addressing unmet medical needs in global health. This decision not only facilitates regulatory approval in the DRC but also lays the groundwork for revising WHO treatment guidelines. Updating the guidelines will expand access across Central and West Africa, ensuring more patients can benefit from this simplified therapy.
Historically, the burden of gambiense sleeping sickness was staggering. In 1998, nearly 40,000 cases were reported, with an estimated 300,000 cases undiagnosed. The only treatment available for late-stage disease was a toxic injectable arsenic derivative. Over the past two decades, innovations like the nifurtimox-eflornithine combination therapy and the first oral treatment, fexinidazole, have progressively improved patient outcomes. In 2024, reported cases fell to fewer than 600, a testament to sustained international collaboration and investment.
Dr. Erick Miaka, Director of the DRC’s national sleeping sickness control programme, highlighted the critical role of local researchers and clinicians. “This positive opinion is a victory for Africa-led science,” he said, noting that rigorous clinical research was conducted in some of the continent’s most remote regions.
Ongoing studies are now examining the safety and effectiveness of Acoziborole Winthrop in children aged 1 to 14. If successful, this could expand the life-saving benefits of the treatment to younger populations and further advance the goal of eliminating gambiense sleeping sickness across Africa.
