EMA Approves First Single-Dose Oral Treatment for African Sleeping Sickness

The European Medicines Agency's human medicines committee (CHMP) has adopted a positive scientific opinion for Acoziborole Winthrop (搜索)搜索该药品, marking a revolutionary advancement in treating human African trypanosomiasis (HAT), commonly known as sleeping sickness. This approval represents the first single-dose oral treatment for the gambiense form of the disease, developed through a collaboration between the Drugs for Neglected Diseases initiative (DNDi) and Sanofi.

Clinical Trial Results Demonstrate High Efficacy

The safety and efficacy of Acoziborole Winthrop (搜索)搜索该药品 are based on results from a single-arm open-label phase II/III clinical trial conducted in the Democratic Republic of Congo and Guinea. The study involved 208 adults and adolescents from 15 years of age with gambiense HAT who received a single dose of the medicine.

After 18 months, treatment was successful in approximately 95% (159 out of 167) of people with second-stage disease and in 100% (41 out of 41) of people with first- and intermediate-stage gambiense HAT. Treatment success was determined by the absence of clinical signs of gambiense HAT, no detectable parasites, and a white blood cell count below a set limit.

Simplified Treatment Approach

Current therapies for gambiense HAT involve multiple doses, intravenous infusions or intramuscular injections, as well as hospitalization. Treatment selection is based on disease stage, which requires determination through a lumbar puncture (spinal tap). Acoziborole Winthrop (搜索)搜索该药品 is administered as a single oral dose and is effective in all stages of gambiense HAT, removing the need for lumbar puncture and hospitalization.

The three-tablet dose can be administered without the need for hospitalization or supervised home care, unlike existing therapies, which require either a 10-day course of oral medication or a combination of injections and oral therapy for advanced cases. This simplification facilitates treatment compliance and accessibility, particularly important for reaching patients in remote areas where the last remaining pockets of the disease persist.

Mechanism of Action and Safety Profile

The exact mechanism by which Acoziborole Winthrop (搜索)搜索该药品 works is not fully understood. However, data suggest that its active substance, acoziborole查看药品详情, binds to and blocks the activity of an enzyme that the parasite needs to produce essential proteins, causing the parasite to die.

The most common side effects reported with Acoziborole Winthrop (搜索)搜索该药品 were headache, fever, weakness, decreased appetite, and tremor. The medicine is contraindicated in people with pre-existing heart rhythm problems and should not be taken with certain medicines used to treat HIV and some other infections. Patients will receive a card explaining these risks, and the company will perform a post-authorization study to collect real-world safety information during the three months after treatment.

Disease Context and Public Health Impact

Gambiense HAT occurs in some parts of Africa and is classified as a neglected tropical disease by the World Health Organization (WHO). The disease is transmitted through the bite of tsetse flies infected with the parasite Trypanosoma brucei gambiense. In the first few days after infection, patients may experience symptoms such as fever, headache, and rash (first stage). Later, they may develop a more severe form affecting the nervous system, causing sleep disturbances and behavioral changes (second stage).

Following concerted control efforts, transmission has been eliminated in many countries. Fewer than 1,000 cases are now reported annually on the African continent, representing a dramatic decline from nearly 40,000 cases reported across Africa in 1998. Experts believe more than 300,000 additional cases were never diagnosed during the peak years. By 2024, that number had fallen to fewer than 600 cases, achieving a remarkable 98% reduction since 2001.

Regulatory Pathway and Access

Acoziborole Winthrop (搜索)搜索该药品 was assessed under the EU-M4all programme, which aims to facilitate patient access in low- and middle-income countries to essential medicines intended to prevent or treat diseases of major public health interest. The application was assessed under an accelerated timetable to enable faster patient access.

EMA's positive scientific opinion will streamline WHO prequalification and facilitate national registration of Acoziborole Winthrop (搜索)搜索该药品 for use in public health programmes in countries where the disease is endemic. Sanofi will donate the drug to WHO for free distribution to patients, supporting the organization's goal of eliminating sleeping sickness by 2030.

Dr. Luis Pizarro, Executive Director at DNDi, emphasized the significance of this advancement: "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. 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."

The treatment is indicated for use in adults and adolescents from 12 years of age weighing at least 40 kg to treat first-stage and second-stage (including severe second-stage) gambiense HAT. Another study currently underway in the Democratic Republic of Congo and Guinea is investigating Acoziborole Winthrop (搜索)搜索该药品 for the treatment of children ages 1 to 14.