Drug name
Last update: Jul 2026Acoziborole
Developer(s)
Drug information
Acoziborole; SCYX-7158; 4‑fluoro‑N‑(1‑hydroxy‑3,3‑dimethyl‑2,1‑benzoxaborol‑6‑yl)‑2‑(trifluoromethyl) benzamide
Acoziborole Winthrop
Small molecule
CPSF3 inhibitor
Acoziborole is a novel benzoxaborole derivative that inhibits the cleavage and polyadenylation specificity factor 3 (CPSF3) enzyme in Trypanosoma brucei parasites. Acoribozole is a long-acting oral therapy with an intrinsic ADME properties that results in a prolonged half life ~272–400 hours. In plasma, 95% of circulating drug is unchanged acoziborole and only small fractions (<3%) are oxidized metabolites. Acoziborole is predominantly released from the body via slow biliary-fecal route. By 60 days, ~87% of total radioactivity has been excreted, mostly in feces (~74%). The drug appeared rapidly in plasma (at 1 h), reached tmax between 24 and 72 h, and remained stable for up to 96 h, after which a slow decrease was quantifiable until 14 weeks after dosing.
Acoziborole Winthrop 960 mg has received positive opinion from European Union including all 27 member states and 3 member states of the European Free Trade Association (EFTA).
Acoziborole Winthrop 960 mg single dose therapy has received positive opinion from European Medicines Agency for treatment of African trypanosomiasis through accelerated assessment programme.
Therapeutic area(s)
- Sleeping sickness
- Treatment
Administration route
Oral
Associated long-acting platforms
Oral solid form
Use of drug
- Self-administered
- Once
Dosage
320 mg tablet
1200 mg
Acoziborole Winthrop 960 mg (320 mg*3 as single dose) is indicated for use in adults and adolescents > 12 years of age weighing at least 40 kg to treat first-stage and second-stage (including severe second-stage) human African trypanosomiasis (sleeping sickness).
The listed dosage information is based on the documents submitted to EMA’s human medicines committee (CHMP) and published literature.
Associated compounds, formulations and regimens
Not provided
Associated technologies
Not provided
Additional information
Developer(s)
Sanofi
Sanofi was established in February 15, 1973 in France as a subsidiary of the oil company Elf Aquitaine,. It has merged with Synthélabo in 1999, then acquired Aventis in 2004, and later Genzyme in 2011. Sanofi Winthrop Industrie is a major pharmaceutical manufacturing subsidiary of the global French biopharmaceutical company Sanofi. It produces a wide range of medications, including small molecules
Drugs for Neglected Diseases Initiative
The Drugs for Neglected Diseases initiative (DNDi) is an international, not‑for‑profit research and development organization. It was established in 2003 through a partnership involving the Indian Council of Medical Research, the Oswaldo Cruz Foundation, KEMRI, the Malaysian Ministry of Health, and Institut Pasteur of France, together with the participation of WHO/TDR and Médecins Sans Frontières.
Drug structure
Scale-up and manufacturing prospects
Not provided
Round-bottom flasks / reaction flasks, 20 mL scintillation vials, Magnetic stirrers / stir bars, Oil bath for heated reactions, Ice bath / cooling setup, Nitrogen / inert atmosphere setup, Sealed reaction vessels with rubber septa / Teflon screw caps, Büchi rotary evaporator for solvent removal under vacuum, Vacuum source / vacuum concentration setup, Drying / concentration setup.
Acoziborole and related compounds can be manufactured in ISO Class 8 (equivalent to EU GMP Grade D “at rest”) by Sanofi Winthrop Industrie. Manufacturing process includes:- 1) Preparation the aryl-amine precursor 2) Install the boron functionality (boronylation) under Pd catalysis at controlled temperature 3) Aldehyde reduction and cyclize to the oxaborole ring 4) Debenzylate / deprotect to obtain the 6-amino oxaborole intermediate 5) Form the final amide (key finishing step for acoziborole-type molecules)
1) HPLC, LCMS, NMR, Silica gel chromatography, 2) Flash chromatography using Teledyne ISCO CombiFlash / Companion systems, 3) Reverse-phase chromatography / Biotage C18 cartridges, 4) Prep-HPLC / Waters Prep LC systems, and 5) Crystallization / isolation after solvent remova
Excipients & delivery device(s)
Not provided
Not provided
No residual solvent used
No delivery device
Safety, Efficacy and Evidence Summary
Not provided
Not provided
Not provided
Not provided
Boron-containing small molecules as anti-protozoal agents
This invention provides, among other things, novel compounds useful for treating protozoal infections, pharmaceutical compositions containing such compounds, as well as combinations of these compounds with at least one additional therapeutically effective agent.
WO2010045503A1
Not provided
Anacor Pharmaceuticals LLC
Not provided
April 15, 2011
Ceased