EMA endorses single-dose acoziborole to eliminate sleeping sickness

UK—The European Medicines Agency’s Committee for Medicinal Products for Human Use has endorsed Acoziborole Winthrop as a single-dose oral treatment for both early and advanced stages of gambiense sleeping sickness.

The approval extends to adults and adolescents aged 12 and older weighing at least 40 kilograms.

This positive opinion demonstrates that the medicine meets rigorous European standards and follows the EU-M4all pathway, which prioritizes therapies addressing diseases with urgent medical needs by consulting with WHO disease experts and regulators from endemic countries.

The endorsement paves the way for regulatory approval in the Democratic Republic of Congo and supports a potential revision of WHO sleeping sickness treatment guidelines.

Consequently, patients across Central and West Africa may gain broader access to the medicine in coming months.

Developed jointly by the Drugs for Neglected Diseases initiative and Sanofi, Acoziborole Winthrop represents a substantial improvement over existing therapies, which require either a 10-day oral regimen or combined injections and oral medications for advanced cases.

From arsenic to innovation

Dr Luis Pizarro, Executive Director at DNDi, emphasized the transformation achieved over two decades.

The field has evolved from complicated treatments involving arsenic derivatives with severe side effects to a single-dose therapy administered over one day.

He attributed this progress to collaborative science and expressed hope that it moves closer to eliminating sleeping sickness, a disease responsible for millions of deaths across Africa over the past century.

Transmitted through infected tsetse fly bites, human African trypanosomiasis—commonly called sleeping sickness—proves almost universally fatal without treatment.

Early-stage symptoms include headaches and fever, while the disease progresses when the parasite crosses the blood-brain barrier and attacks the central nervous system, triggering behavioral changes, cognitive impairment, neurological complications, seizures, sleep disruption, aggression, confusion, lethargy, and ultimately death.

Clinical evidence and regional impact

DNDi conducted a pivotal Phase II/III study across the Democratic Republic of Congo and Guinea in collaboration with national sleeping sickness programs, while Sanofi managed the regulatory approval process.

The clinical trial, published in The Lancet Infectious Diseases, demonstrated efficacy rates of up to 96 percent across both disease stages with an acceptable safety profile.

Dr Erick Miaka, Director of the DRC’s national sleeping sickness control programme, described the development as a victory for African-led science, crediting African doctors and researchers who conducted rigorous pharmaceutical research in remote regions.

Progress against a devastating disease

Since 1998, when nearly 40,000 cases were reported alongside an estimated 300,000 undiagnosed cases, significant progress has emerged.

A half-century of investment in new therapies brought nifurtimox-eflornithine combination therapy in 2009 and fexinidazole in 2018.

By 2024, cases fell below 600 annually, representing a 98 percent reduction since 2001.

Audrey Duval, Executive Vice President of Corporate Affairs at Sanofi, highlighted the company’s sustained commitment to fighting sleeping sickness through partnerships with DNDi, the WHO, and other organizations.

Sanofi will donate Acoziborole Winthrop to the WHO through its philanthropic organization, Foundation S – The Sanofi Collective, ensuring free access for patients.

Additionally, researchers are investigating the treatment’s effectiveness in children aged 1 to 14 through ongoing studies in the Democratic Republic of Congo and Guinea.