NAIROBI, Kenya Feb 27 – A single-dose pill to treat sleeping sickness has cleared a major regulatory milestone, raising fresh hopes of eliminating one of Africa’s deadliest parasitic diseases.The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion for Acoziborole Winthrop (acoziborole), a one-day oral treatment for both early and advanced stages of gambiense human African trypanosomiasis in adults and adolescents aged 12 and above weighing at least 40 kilogrammes.The decision was granted under the EU-M4all procedure, a pathway designed for high-priority medicines targeting diseases with unmet medical needs outside the European Union.The process involves experts from the World Health Organization and regulators from endemic countries, offering a strong scientific endorsement that the drug meets EU standards for safety, efficacy and quality.The ruling is expected to support regulatory approval in the Democratic Republic of the Congo the country most affected by the disease and pave the way for revisions to WHO treatment guidelines.An update to the guidelines would enable broader rollout across Central and West Africa, where the disease remains endemic.Acoziborole was co-developed by the Drugs for Neglected Diseases initiative and Sanofi. If approved locally, it would represent a major advance over current therapies, which require either a 10-day course of oral medication or a combination of injections and tablets for advanced-stage patients.“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 Dr Luis Pizarro, Executive Director of DNDi.He described the development as a testament to collaborative science and a crucial step toward eliminating a disease that has killed millions across Africa over the past century.Sleeping sickness is transmitted by the bite of an infected tsetse fly and is almost always fatal if untreated.Early symptoms include fever and headaches. In later stages, the parasite crosses the blood-brain barrier and invades the central nervous system, causing severe neurological and behavioural symptoms including confusion, aggression, seizures, sleep disturbances and, ultimately, death.The CHMP’s positive opinion is based on clinical and non-clinical data, including findings from a pivotal Phase II/III trial conducted in the Democratic Republic of the Congo and Guinea in partnership with national sleeping sickness control programmes.The study, published in The Lancet Infectious Diseases, showed success rates of up to 96 per cent at 18 months across both stages of the disease, with a favourable safety profile.“The development of acoziborole and today’s positive scientific opinion is a victory for Africa-led science,” said Dr Erick Miaka, Director of the DRC’s national sleeping sickness control programme.He credited African doctors and researchers who conducted trials in some of the continent’s most remote areas.The fight against gambiense sleeping sickness has made dramatic progress over the past two decades.In 1998, nearly 40,000 cases were reported, with an estimated 300,000 undiagnosed. At the time, late-stage treatment relied on injectable arsenic derivatives associated with serious side effects.Subsequent innovations included the nifurtimox-eflornithine combination therapy introduced in 2009 and fexinidazole the first all-oral treatment approved in 2018.By 2024, reported cases had dropped to fewer than 600, representing a 98 per cent reduction since 2001.Sanofi said it will donate Acoziborole Winthrop to the WHO through its philanthropic arm, Foundation S – The Sanofi Collective, ensuring the medicine is provided free of charge to patients in endemic countries.A separate study is now underway in the Democratic Republic of the Congo and Guinea to assess the treatment’s safety and effectiveness in children aged one to 14.