NAIROBI, Kenya, Mar 2 – The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion for Acoziborole Winthrop (acoziborole), a new single-dose oral treatment for gambiense human African trypanosomiasis, commonly known as sleeping sickness.
The drug is designed as a three-tablet, one-time treatment for both early and advanced stages of the disease. It is intended for adults and adolescents aged 12 years and older who weigh at least 40 kilograms, subject to approval by affected countries.
Acoziborole was co-developed by the Drugs for Neglected Diseases initiative (DNDi) and Sanofi.
Sleeping sickness is transmitted through the bite of an infected tsetse fly and is almost always fatal if left untreated. Early symptoms include headaches and fever. In later stages, the disease affects the nervous system, leading to behavioural changes, confusion, seizures, sleep disturbances and eventually death.
The positive opinion is based on clinical and non-clinical data, including results from a Phase II/III study published in The Lancet Infectious Diseases. The study showed success rates of up to 96 percent after 18 months across both stages of the disease, with a strong safety profile.
Because the medicine is not intended for sale in Europe, the EMA will not formally register it. Instead, it issued the positive opinion under a special procedure known as Medicines4all. This framework allows countries where the disease is common to rely on the EMA’s scientific assessment when approving the drug locally.
DNDi Executive Director Luis Pizarro said current treatments require either a 10-day course of oral medicine or a combination of injections and oral therapy for advanced cases. He expressed optimism that the new single-dose treatment will offer a simpler and safer alternative.
The positive opinion is expected to support regulatory approvals in endemic countries, particularly in Central and West Africa, and may also lead to updates in World Health Organization treatment guidelines.
Sanofi announced that it will donate Acoziborole Winthrop to the WHO through its philanthropic arm, Foundation S – The Sanofi Collective, making the treatment available free of charge to patients in affected countries.
Company officials said the milestone reflects strong collaboration between the partners and represents a significant step toward eliminating sleeping sickness.
