Chemistry:Acoziborole
Acoziborole (SCYX-7158) is an antiprotozoal drug invented by Anacor Pharmaceuticals in 2009,[1] and now under development by the Drugs for Neglected Diseases Initiative with Sanofi for the treatment of African trypanosomiasis (sleeping sickness).
It is a structurally novel drug described as a benzoxaborole derivative, and is a one-day, one-dose oral treatment. Phase I human clinical trials were completed successfully in 2015.[2] A single arm phase II/III trial, with no control group, was conducted from 2016 to 2019 in the Democratic Republic of the Congo and Guinea involving 208 eligible patients with trypanosomiasis caused by Trypanosoma brucei gambiense. The results of the study, published in The Lancet on 29 November 2022, found the treatment regimen had an efficacy greater than 95%. A trial in paediatric patients was ongoing in 2023.[3]
As the regimen is significantly easier to administer compared to existing treatment options, some commentators expressed hope that acoziborole could significantly slow down or even eliminate the transmission of African trypanosomiasis in humans.[4][5] To determine whether acoziborole could be used to address the reservoir of T.b. gambiense, in support of the World Health Organization objective to interrupt disease transmission by 2030, a double-blind, randomized trial of the drug in 1,200 seropositive non-parasitologically confirmed participants was completed in 2023.[6]
The European Medicines Agency adopted a positive scientific opinion on acoziborole in February 2026, paving the way for registration and distribution in endemic countries[7].
In 2016, Drugs for Neglected Diseases initiative anticipated that the cost of developing and gaining approval for acoziborole would be around $50 million.[8]
Acoziborole was approved by the European Medicines Agency in February 2026.[9]
See also
References
- ↑ "SCYX-7158, an orally-active benzoxaborole for the treatment of stage 2 human African trypanosomiasis". PLOS Neglected Tropical Diseases 5 (6). June 2011. doi:10.1371/journal.pntd.0001151. PMID 21738803.
- ↑ "DNDi announces successful completion of SCYX-7158 Phase I study for treatment of sleeping sickness". 9 September 2015. http://www.news-medical.net/news/20150909/DNDi-announces-successful-completion-of-SCYX-7158-Phase-I-study-for-treatment-of-sleeping-sickness.aspx.
- ↑ "ACOZI-KIDS | DNDi". 26 November 2021. https://dndi.org/global-networks/acozi-kids/.
- ↑ "Single dose of acoziborole 95% effective in treating sleeping sickness". November 30, 2022. https://www.cidrap.umn.edu/news-perspective/2022/11/single-dose-acoziborole-95-effective-treating-sleeping-sickness.
- ↑ "Sleeping sickness: time for dreaming". The Lancet. Infectious Diseases 23 (4): 387–388. April 2023. doi:10.1016/S1473-3099(22)00686-7. PMID 36460028.
- ↑ "Acoziborole | DNDi". 31 December 2009. https://dndi.org/research-development/portfolio/acoziborole/.
- ↑ "New single-dose oral treatment for human African trypanosomiasis (sleeping sickness) | European Medicines Agency (EMA)" (in en). 2026-02-27. https://www.ema.europa.eu/en/news/new-single-dose-oral-treatment-human-african-trypanosomiasis-sleeping-sickness.
- ↑ (in en) Busting the Billion-Dollar Myth: How to Slash the Cost of Drug Development. Nature Magazine. PMID 27558048. https://www.scientificamerican.com/article/busting-the-billion-dollar-myth-how-to-slash-the-cost-of-drug-development/. Retrieved 2025-06-13.
- ↑ "'Truly spectacular' drug for sleeping sickness simplifies treatment, raising hopes for eradication". Science 391 (6789): 962–963. 2026. doi:10.1126/science.aeg9251. PMID 41785354. https://www.science.org/content/article/truly-spectacular-drug-sleeping-sickness-simplifies-treatment-raising-hopes-eradication. Retrieved 17 March 2026.
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