Africa’s long battle against sleeping sickness may be approaching its final chapter as a new single-dose therapy gains international regulatory backing.
The drug, acoziborole, received a positive scientific opinion on February 27 from the Committee for Medicinal Products for Human Use of the European Medicines Agency, clearing the way for national approval processes in countries where the disease remains endemic.
The decision was issued under the EU-M4all programme, a regulatory pathway designed to accelerate access to medicines intended primarily for use outside the European Union.
Under this procedure, scientific assessment by European regulators supports approvals in countries facing major public health challenges.
Public health experts say the simplicity of the treatment could transform the fight against sleeping sickness, enabling front-line teams to diagnose and treat patients in a single encounter. “The simplicity of acoziborole will allow health workers to test and cure patients in the same visit, which is a game changer for rural programs,” said Erick Miaka, head of the National Sleeping Sickness Control Programme in the Democratic Republic of Congo.
Sleeping sickness, formally known as human African trypanosomiasis, is a parasitic disease transmitted by the bite of infected tsetse flies.
The illness attacks the central nervous system and is almost always fatal if left untreated. For much of the past century, treating the disease often meant confronting an impossible choice.
The only available therapy for advanced infections was an arsenic-based injection so toxic that it killed roughly five percent of patients who received it, according to the Drugs for Neglected Diseases initiative. Doctors administered the treatment because the alternative was certain death. Untreated infections gradually invade the brain, causing neurological damage, severe sleep disruptions, confusion, and eventually coma.
Over the past two decades, however, a coordinated campaign involving African governments, research institutions, international health organisations and pharmaceutical partners has steadily reduced the disease’s footprint.
With acoziborole, patients will no longer have to endure a 10-day course of treatment that previously created logistical challenges for health workers operating in remote areas. The medicine is administered as a single dose of three tablets.
Evidence supporting the therapy comes from a Phase II/III clinical study conducted in the Congo and Guinea.
The trial enrolled 208 adult and adolescent patients and followed them for 18 months. The results showed treatment success rates of about 95% among patients with late-stage disease and 100% among those in earlier stages, according to the European Medicines Agency’s scientific assessment.
Those findings have raised hopes that the drug could accelerate Africa’s progress toward eliminating the disease entirely. Human African trypanosomiasis exists in two forms, but the Gambiense variant accounts for more than 90% of infections worldwide and is found primarily in West and Central Africa.
Unlike many infectious diseases, humans are the main reservoir for this form of the parasite. That biological characteristic means elimination becomes achievable if transmission is interrupted and infected individuals are treated quickly.
The development of acoziborole also highlights Africa’s growing role in clinical research for neglected diseases. Trials for the drug were conducted in partnership with African research institutions and national disease control programmes.
The programme behind acoziborole was led by the Drugs for Neglected Diseases initiative in collaboration with Sanofi, which has pledged to donate the medicine for use in endemic countries through the World Health Organisation’s distribution mechanisms. – Bird Story Agency
