EMA CHMP grants positive opinion to DNDi/Sanofi’s single-dose Acoziborole Winthrop for gambiense sleeping sickness in adults/adolescents. 96% efficacy at 18 months surpasses multi-day regimens, paving way for DRC approval and WHO guideline updates to eliminate this fatal tsetse-transmitted disease.
Written By: Chikkula Pavan Kumar, PharmD
Reviewed By: Pharmacally Editorial Team
The European Medicines Agency‘s CHMP has issued a positive opinion for Acoziborole Winthrop (acoziborole), a groundbreaking single-dose oral therapy targeting both early- and advanced-stage gambiense human African trypanosomiasis commonly known as sleeping sickness in adults and adolescents aged 12+ weighing at least 40 kg.
This accelerated assessment, designed for high-unmet-need medicines outside the EU, clears a path for approval in the Democratic Republic of Congo (DRC) and updates to WHO guidelines, expanding access across Central and West Africa.
Co-developed by the Drugs for Neglected Diseases initiative (DNDi) and Sanofi, acoziborole promises to eclipse current regimens: a 10-day oral course or injection-oral combos for late-stage cases.
Human African trypanosomiasis, or gambiense sleeping sickness, earned its name from hallmark late-stage symptoms where the Trypanosoma brucei gambiense parasite crosses the blood-brain barrier, disrupting the sleep-wake cycle with profound lethargy, nocturnal insomnia, and daytime somnolence alongside cognitive decline.
Caused by the protozoan parasite and transmitted via tsetse fly bites in Central/West Africa, it progresses silently from a first-stage chancre, fever, and lymphadenopathy to second-stage meningoencephalitis, marked by personality changes, seizures, coma or death if untreated
Acoziborole counters this by exploiting the parasite’s unique adenosine transporter (TbAT1) for selective uptake, followed by rapid conversion to a toxic purine analog that inhibits DNA synthesis and glycolysis, achieving pan-stage efficacy in a single oral dose without arsenic toxicity.
The CHMP opinion is supported by clinical and non-clinical data, including a pivotal Phase 2/3 trial (NCT03087955) conducted in the Democratic Republic of Congo and Guinea in partnership with national sleeping sickness control programs. The study, published in The Lancet Infectious Diseases, demonstrated treatment success rates of up to 96% at 18 months across both early and late stages of the disease, with a favorable safety profile.
An additional study is ongoing to evaluate the medicine in children aged 1 to 14 years.
“This single-dose therapy marks 20 years of progress from arsenic-laced injections to safe, one-day cures,” said DNDi Executive Director Luis Pizarro, MD.
Erick Miaka, MD, DRC National Sleeping Sickness Control Program Director, hailed it as “a victory for Africa-led science” from remote trials.
From 300,000 estimated cases in 1998 to under 600 in 2024, innovations like 2009’s nifurtimox-eflornithine combo (NECT) and 2018’s 10-day oral fexinidazole slashed incidence 98% since 2001.
Sanofi, committing to donation via WHO and its Foundation S, eyes elimination by 2030.
Backed by global funders, DNDi’s acoziborole optimized from a Pfizer-acquired hit joins 13 prior treatments, underscoring public-private collaboration’s power against neglected diseases.
Reference:
Acoziborole Winthrop, developed by DNDi and Sanofi, receives CHMP positive opinion as three-tablet, single-dose treatment for most common form of sleeping sickness
https://www.sanofi.com/en/media-room/press-releases/2026/2026-02-27-10-55-51-3246379
European Medicines Agency (EMA) Positive scientific opinion information, New single-dose oral treatment for human African trypanosomiasis (sleeping sickness)
Betu Kumeso, Victor Kande et al., Efficacy and safety of acoziborole in patients with human African trypanosomiasis caused by Trypanosoma brucei gambiense: a multicentre, open-label, single-arm, phase 2/3 trial, The Lancet Infectious Diseases, Volume 23, Issue 4, 463 – 470, https://doi.org/10.1016/S1473-3099(22)00660-0
Efficacy and Safety of Acoziborole (SCYX-7158) in Patients with Human African Trypanosomiasis Due to T.b. Gambiense (OXA002), ClinicalTrials.gov ID NCT03087955, https://clinicaltrials.gov/study/NCT03087955
Trypanosomiasis, human African (sleeping sickness), 02 May 2023, https://www.who.int/news-room/fact-sheets/detail/trypanosomiasis-human-african-(sleeping-sickness)
Acoziborole, DNDI, 27 February 2026, https://dndi.org/research-development/portfolio/acoziborole/
About Writer
Chikkula Pavan Kumar, Pharm.D.
He is a Doctor of Pharmacy with a keen interest in clinical pharmacy, pharmacovigilance, and evidence-based practice. In his words, he is passionate about patient safety and translating complex medical information into clear, research-driven communication.