Come 2019, countries where sleeping sickness is endemic will see distribution of fexinidazole, the first all-oral treatment for the disease.
According to experts, Fexinidazole will support international efforts to eliminate sleeping sickness, a fatal neglected tropical disease endemic to Africa.
The new drug has been recommended by European Medicines Agency’s committee for Medicinal products for Human Use (CHMP) and has shown the drug to be efficacious for both stages of sleeping sickness.
This approval is a result of clinical trials led by the non-profit research and development organization (DNDi) and an application submitted by Sanofi.
DNDi is a not-for-profit research and development organisation which works to deliver new treatments for neglected diseases, in particular human African trypanosomiasis, leishmaniasis, Chagas disease, filarial infections, mycetoma, paediatric HIV, and hepatitis C while, Sanofi is a global biopharmaceutical company focused on human health and prevention of illness with vaccines.
Sleeping sickness, or human African trypanosomiasis (HAT), is usually fatal without treatment. About 65 million people in sub-Saharan Africa are at risk.
Transmitted by the bite of a tsetse fly, it causes neuropsychiatric symptoms; including aggression, psychosis, and a debilitating disruption of sleep patterns that have given this neglected disease its name.
“I’ve dedicated my life as a doctor to sleeping sickness. An all-oral treatment has been a dream of mine for decades.
Those affected are some of the most vulnerable and live in some of the most remote areas of the Congo, if not the world. They need a treatment that is safe, effective and simple,” said Dr Victor Kande.
Kande ,Neglected Tropical Diseases Expert Advisor to the Ministry of Health of the Democratic Republic of Congo (DRC), was the principal investigator of the trials.
“Less than ten years ago we were still treating this disease with an arsenic derivative that killed 5% of all patients.
While current treatments are safe and effective, they require a patient to be hospitalised and pose a huge logistical burden on the health system. Fexinidazole comes as a simple pill: this is a huge leap in how we can tackle this deadly disease,”he added
Fexinidazole is indicated as a 10-day once-a-day treatment for Trypanosoma brucei gambiense sleeping sickness (the most common form of the disease, found in West and Central Africa).
Importantly, fexinidazole is the first all-oral treatment that works both for the early stage of the disease as well as the second stage of the disease in which the parasites have crossed the blood-rain barrier, causing patients to suffer from neuropsychiatric symptoms.
During the clinical trials, which enrolled 749 patients in the DRC and Central African Republic, fexinidazole showed high efficacy and safety in both stages of the disease, both in adults and children aged six years and weighing 20 kg.
Results showed that fexinidazole could, therefore, eliminate the need for systematic hospitalisation and potential reduction in number of lumbar punctures.
“Fexinidazole is an entirely new chemical entity that has been developed through an alternative non-profit R&D model.
It is the first new chemical entity to be developed by DNDi,” said Dr Bernard Pécoul, DNDi Executive Director. “This therapeutic breakthrough is testament to the unique partnership between DNDi and Sanofi to discover, develop, and register a treatment for a severely neglected disease.”
“This therapeutic breakthrough is the latest milestone in Sanofi’s long-term commitment to sleeping sickness,” said Dr Ameet Nathwani, Chief Medical Officer and Executive Vice President Medical Function.
“Fexinidazole is the proof that partnerships between public and private sectors can deliver safe and effective medicines for the most neglected patients. Sanofi is proud to donate this medicine to the World Health Organisation as part of our mission to support the elimination of sleeping sickness.”
Current disease distribution
According to available data from WHO, the disease incidence differs from one country to another as well as in different parts of a single country.
In the last 10 years, over 70% of reported cases occurred in the Democratic Republic of the Congo (DRC).
The DRC is the only country that currently reports more than 1000 new cases annually and accounts for 84% of the cases reported in 2015.
Central African Republic is the only country that declared between 100 and 200 new cases in 2015.
Countries such as Angola, Burkina Faso, Cameroon, Chad, Congo, Côte d'Ivoire, Equatorial Guinea, Gabon, Gjana, Guinea, Malawi, Nigeria, South Sudan, Uganda, United Republic of Tanzania, Zambia and Zimbabwe are reporting fewer than 100 new cases per year.