Acoziborole Winthrop developed by Sanofi officially receives positive opinion as three-tablet, single-dose treatment for the most common form of sleeping sickness

The EMA’s CHMP has recently granted a positive opinion to Acoziborole Winthrop as a single-dose oral treatment for both early and advanced-stage gambiense sleeping sickness in adults as well as in adolescents 12 years and older weighing at least 40 kilograms.

This positive opinion is granted through accelerated assessment under a specific procedure intended for countries outside of the European Union and used for high-priority medicines for diseases with unmet needs. This will facilitate approval of the medicine in the Democratic Republic of Congo and pave the way for an update of WHO’s sleeping sickness treatment guidelines, a move that would eventually expand access to other countries in Central and West Africa, where the disease is endemic.

Note that transmitted by the bite of an infected tsetse fly, human African trypanosomiasis, commonly known as sleeping sickness, is almost always fatal without treatment. In the early stage of the disease, people experience headaches or fever. In the late stage, the parasite crosses the blood-brain barrier and invades the central nervous system, causing behavioral, cognitive and neurological symptoms, including seizures, sleep disturbances, aggression, confusion, lethargy, convulsions, and, ultimately, death.

“In just 20 years, we have gone from complicated treatments including arsenic derivatives with serious side effects, to today, when a single-dose, one-day therapy could safely cure patients,” said Luis Pizarro, MD, Executive Director at DNDi. “This progress is testament to the transformative power of collaborative science and will bring us closer to finally eliminating sleeping sickness, a disease that has killed millions on the African continent in the past century.”

In addition, DNDi has conducted a pivotal phase 2/3 study in the DRC and Guinea in partnership with National Sleeping Sickness Control Programs, while Sanofi carried out the regulatory approval process. The CHMP positive opinion is based on clinical and non-clinical data provided by the partners, with efficacy and safety supported by the phase 2/3 study.

“The development of acoziborole and today’s positive scientific opinion is a victory for Africa-led science, made possible thanks to African doctors and researchers who conducted cutting-edge pharmaceutical research in some of the most remote and difficult-to-reach areas on the continent,” said Erick Miaka, MD, Director of the DRC’s national sleeping sickness control program.

“For decades, Sanofi has maintained an unwavering commitment to the fight against sleeping sickness, standing alongside DNDi, the World Health Organization, and other partners in one of the most enduring and successful public-private health collaborations,” said Audrey Duval, Executive Vice President, Corporate Affairs at Sanofi. “Together, we have helped drive cases to historic lows—achieving a remarkable 98% reduction since 2001—by putting patients first and investing in innovation where it is needed most. Acoziborole builds on this legacy and represents a decisive step forward in eliminating gambiense sleeping sickness by 2030.”

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Acoziborole Winthrop, CHMP, dndi, Sanofi, sickness, sleeping sickness