The European Medicines Agency has given the green light to the first-ever single-dose oral treatment for sleeping sickness.
Acoziborole Winthrop, developed by the Drugs for Neglected Diseases initiative (DNDi) in collaboration with Sanofi, will help African countries eliminate the most common form of the disease, gambiense sleeping sickness.
A positive CHMP opinion through the EU-M4all procedure provides a strong endorsement that the
medicine meets EU standards.
The breakthrough follows clinical trials in the Democratic Republic of the Congo and Guinea led by African researchers. Acoziborole Winthrop (acoziborole) will treat both early and advanced stages of the disease in adults, as well as adolescents aged 12 years and older weighing at least 40 kilograms.
The three-tablet dose can be administered without the need for hospitalisation or supervised home care, unlike existing therapies, which require either a 10-day course of oral medication or a combination of injections and oral therapy for advanced cases.
DNDi hailed the milestone, saying it offers a simpler alternative which will help support the World Health Organisation’s goal of eliminating the disease by 2030.
“ In just 20 years, we have gone from complicated treatments including arsenic derivatives with serious side effects, to today, when a single-dose, one-day therapy could safely cure patients,’ said Dr Luis Pizarro, Executive Director at DNDi.
“This progress is testament to the transformative power of collaborative science and will bring us closer to finally eliminating sleeping sickness, a disease that has killed millions on the African continent in the past century”, he added.
Acoziborole will allow doctors to reach the most remote areas, where the last remaining pockets of the disease are. In 1998, nearly 40,000 cases were reported across Africa, and experts believe more than 300,000 others were never diagnosed. By 2024, that number had fallen to fewer than 600 cases.
Sanofi, which will donate the drug to WHO for free distribution to patients, described the development as a major step forward in eradicating the disease, which remains a public health problem, particularly in Central and West Africa.
“For decades, Sanofi has maintained an unwavering commitment to the fight against sleeping sickness, standing alongside DNDi, the World Health Organization, and other partners in one of the most enduring and successful public-private health collaborations,’ said Audrey Duval, Executive Vice President, Corporate Affairs at Sanofi.
“Together, we have helped drive cases to historic lows— achieving a remarkable 98% reduction since 2001—by putting patients first and investing in innovation where it is needed most. Acoziborole builds on this legacy and represents a decisive step forward in eliminating gambiense sleeping sickness by 2030”, Duval added.
Another study underway in the DRC and Guinea is investigating Acoziborole Winthrop for the treatment of children ages 1 to 14.
EXPLAINER: How is the disease transmitted?
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.
