Acoziborole

Acoziborole (SCYX-7158) is an antiprotozoal drug invented by Anacor Pharmaceuticals in 2009, 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. 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.

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. 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.

The European Medicines Agency adopted a positive scientific opinion on acoziborole in February 2026, paving the way for registration and distribution in endemic countries.

In 2016, Drugs for Neglected Diseases initiative anticipated that the cost of developing and gaining approval for acoziborole would be around $50 million.

Acoziborole was approved by the European Medicines Agency in February 2026.

• Tavaborole