Acoziborole: A Single-Dose Breakthrough in Sleeping Sickness Treatment

A new drug, acoziborole, has received an important approval from a committee of the European Medicines Agency. This medication is notable as the first single-dose treatment for sleeping sickness, a disease caused by a parasite transmitted by the tsetse fly. Administered as three pills taken together in a single dose, acoziborole's development strongly supports the World Health Organization's (WHO) objective to eliminate sleeping sickness by 2030.

A Breakthrough in Treatment: Comparing Old and New

Previous treatments for sleeping sickness presented significant challenges:

• Older intravenous drugs caused a "burning" sensation and were fatal for nearly one in 20 patients.
• The current first-line oral treatment, fexinidazole, requires a 10-day course and is associated with severe side effects such as nausea, vomiting, and heart-rhythm disturbances.

Dr. Stéphane Hugonnet, involved in the clinical trials, noted that the harsh side effects of past medications discouraged individuals from seeking treatment.

In stark contrast, clinical trials for acoziborole identified only one significant side effect: mild to moderate headache. This dramatically simpler and safer profile marks a major improvement in patient care.

Dr. Gerardo Priotto, who leads the WHO's sleeping sickness efforts, stated that previous therapies were difficult to use, requiring specific staff, equipment, and reliable infrastructure, which were often lacking in remote, rural areas where most cases occur.

Impact on Elimination Goals and Future Directions

Acoziborole simplifies patient care and improves treatment access, accelerating progress toward the elimination of sleeping sickness, according to Priotto. The drug treats both early and late stages of the disease, addressing the Trypanosoma brucei gambiense parasite, which accounts for over 90% of cases.

Due to sustained global efforts, sleeping sickness has seen a drastic reduction in incidence, with approximately 1,000 cases annually. Nearly two-thirds of these cases are concentrated in the Democratic Republic of Congo (DRC).

The Drugs for Neglected Diseases Initiative (DNDi), a nonprofit, developed acoziborole with pharmaceutical company Sanofi and crucial funding from partners including the Gates Foundation. Dr. Wilfried Mutombo Kalonji, head of West and Central Africa Clinical Operations for DNDi, highlighted the inherent challenges of conducting clinical trials in remote areas, particularly due to a lack of basic infrastructure.

Currently, a new trial is underway to assess if simple blood (serologic) tests can reliably allow immediate treatment. This has the potential to significantly increase the number of treated patients and help stop transmission by eliminating human reservoirs of the parasite.

The next stages for acoziborole include reviews by the Democratic Republic of Congo Ministry of Health and the WHO to update treatment guidelines. These updates would facilitate the drug's authorization and distribution in other countries where it is needed. However, concerns exist regarding potential future funding cuts from U.S. and Western sources that could impede the drug's availability in countries needing it most.