The US Food and Drug Administration (FDA) has approved fexinidazole as the first all-oral treatment for both stages of sleeping sickness (human African trypanosomiasis).
Fexinidazole was developed as part of an innovative partnership between the non-profit research and development organization Drugs for Neglected Diseases initiative (DNDi), which conducted the pivotal clinical trials for this treatment, in partnership with the national sleeping sickness programmes of the Democratic Republic of Congo (DRC) and Central African Republic (CAR), and Sanofi.
Sleeping sickness is a parasitic disease transmitted by the bite of an infected tsetse fly. It mostly affects populations living in remote rural areas of sub-Saharan Africa, where about 65 million people are at risk. Left untreated, sleeping sickness is almost always fatal.
Current treatment options for the disease are effective, but burdensome for patients and health workers due to the need for infusion or injection, requiring hospitalization, especially challenging for people living in remote areas.
‘Having a simple all-oral treatment for sleeping sickness is a dream come true for frontline clinicians,’ said Dr Bernard Pécoul, DNDi Executive Director. ‘We are proud of this latest milestone in our long-term partnership with Sanofi, developed in close collaboration with researchers in countries hard-hit by sleeping sickness.’
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. Fexinidazole is the first all-oral treatment that works both for the first stage of the disease, as well as the second stage of the disease in which the parasites have crossed the blood-brain barrier, causing patients to suffer from neuropsychiatric symptoms. Today’s US FDA approves its use in patients ≥ 6 years of age and older and weighing at least 20 kg.
‘This FDA approval is a key milestone in Sanofi’s long-term commitment to fight sleeping sickness, started 20 years ago alongside the WHO through an ambitious partnership to combat Neglected Tropical Diseases,’ said Luc Kuykens, Senior Vice President, Sanofi Global Health unit. ‘Following the positive scientific opinion granted by the European Medicines Agency end 2018, the FDA approval is an important step to revitalize efforts to support the sustainable elimination of the disease.’
As a result of FDA approval, a priority review voucher (PRV) has been awarded to DNDi. The FDA Tropical Disease PRV Program was established in 2007 to incentivize development of new treatments for neglected tropical diseases, including sleeping sickness. Any benefits from the PRV will be shared between Sanofi and DNDi, which will enable continued investments in innovative and accessible new health tools for sleeping sickness and other neglected diseases. Sanofi commits to continue to provide the drug free-of-charge to the World Health Organization (WHO) for distribution to affected countries, as part of a long-term collaboration with WHO.
DNDi, Sanofi and partners are deeply committed to ensuring access to fexinidazole in all sleeping sickness-endemic countries.
About sleeping sickness
Sleeping sickness, or human African trypanosomiasis (HAT), is usually fatal without treatment. Transmitted by the bite of an infected tsetse fly, following a period with nonspecific symptoms, it evolves to cause neuropsychiatric symptoms including abnormal behaviour and a debilitating disruption of sleep patterns that have given this neglected disease its name. About 65 million people in sub-Saharan Africa are at moderate to very high risk of infection.
The Drugs for Neglected Diseases initiative (DNDi) is a collaborative, patient needs-driven, not-for-profit research and development (R&D) organization that develops safe, effective, and affordable treatments for sleeping sickness, leishmaniasis, Chagas disease, filarial infections, mycetoma, paediatric HIV, hepatitis C, and COVID-19. Since its inception in 2003, DNDi has delivered nine new treatments, including nifurtimox-eflornithine combination therapy (NECT) for late-stage sleeping sickness, and fexinidazole, the first all-oral drug for sleeping sickness.
Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions. With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.
Frédéric Ojardias (Geneva)
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Ilan Moss (New York)
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DNDi’s fexinidazole programme is supported by grants from:
DRC – Ministry of Health of the Democratic Republic of Congo (through the Projet de Développement du Système de Santé (PDSS) funded by the World Bank)
European Union – European and Developing Countries Clinical Trials Partnership Association (EDCTP2 Programme)
France – French Development Agency (AFD) and Ministry for Europe and Foreign Affairs (MEAE)
Germany – Federal Ministry of Education and Research (BMBF) through KfW; and the German Corporation for International Cooperation (GIZ) on behalf of the Federal Republic of Germany
the Netherlands – Dutch Ministry of Foreign Affairs (DGIS)
Norway – Norwegian Agency for Development Cooperation (Norad), Norwegian Ministry of Foreign Affairs, as part of Norway’s in-kind contribution to EDCTP2
Spain – Spanish Agency for International Development and Cooperation (AECID)
Switzerland – Republic and Canton of Geneva, Internal Solidarity Service; and the Swiss Agency for Development and Cooperation (SDC)
UK – UK aid
Bill & Melinda Gates Foundation; Brian Mercer Trust; the ELMA Foundation; Else Kröner-Fresenius-Stiftung; Médecins sans Frontières; Stavros Niarchos Foundation; Takeda Pharmaceutical Company Limited; UBS Optimus Foundation; and other private foundations and individuals.
Photo credit: Kenny Mbala-DNDi