Sanofi's Fexinidazole: First All-Oral Therapy for Human African Trypanosomiasis Approved in Democratic Republic of Congo

Recently, the Democratic Republic of the Congo (DRC) approved Sanofi’s fexinidazole for the treatment of African trypanosomiasis (HAT, commonly known as sleeping sickness) caused by Trypanosoma brucei gambiense. This marks the first all-oral therapy approved for sleeping sickness. The approval paves the way for the drug’s authorization in other developing countries. Currently, a marketing authorization application for fexinidazole is planned for submission in Uganda.

Sleeping sickness is usually fatal and has long lacked effective treatments. Transmitted primarily through the bite of the tsetse fly, it causes neurological symptoms, including aggressive behavior, psychiatric abnormalities, and sleep disturbances. Approximately 65 million people in sub-Saharan Africa are at risk of contracting this disease. “I have a deep personal connection to sleeping sickness, having grown up in East Africa where my mother constantly worried about its impact on our family,” said Dr. Ameet Nathwani, Chief Medical Officer and Executive Vice President of Sanofi. “The approval of fexinidazole in the Democratic Republic of the Congo has given Sanofi great hope, and we will strive to eliminate the burden of sleeping sickness next year.”

The majority of sleeping sickness patients worldwide reside in the Democratic Republic of the Congo, followed by the Central African Republic, Guinea, and Chad. The latest data released by the World Health Organization (WHO) in July 2018 confirmed a continued decline in new cases of the disease. In 2017, only 1,447 new cases were reported to the WHO, compared with 2,164 in 2016 and 9,870 in 2009. In 2017, 85% of sleeping sickness patients presented with somnolence symptoms. Although current treatment regimens for sleeping sickness are effective, they impose a substantial burden on both patients and healthcare workers. These regimens require hospitalization and entail long-term logistical challenges, particularly limiting access to these therapeutic benefits for individuals living in remote areas.

The Republic of the Congo has approved fexinidazole as a once-daily oral treatment for sleeping sickness, with a treatment duration of only 10 days. Fexinidazole is indicated for both the early stage of the disease and the second stage, when parasites have crossed the blood-brain barrier. Patients with second-stage sleeping sickness suffer from severe conditions and neurological symptoms, while fexinidazole eliminates the need for hospitalization in all patient types.

Fexinidazole was developed through a collaboration between Sanofi and the Drugs for Neglected Diseases initiative (DNDi). This drug, a 5-nitroimidazole derivative, was initially developed by Hoechst in the 1980s but was later abandoned due to strategic reasons. During DNDi’s search for compounds with antiparasitic activity, fexinidazole was identified in 2005 in partnership with the Swiss Tropical and Public Health Institute as a potential treatment for human African trypanosomiasis caused by Trypanosoma brucei gambiense. Subsequently, DNDi secured the patent rights to fexinidazole, and Sanofi resumed research on the drug for the treatment of sleeping sickness.

On November 16, 2018, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended the approval of fexinidazole for the treatment of sleeping sickness. The company noted that this drug is the first oral therapy effective against both stages of African trypanosomiasis (sleeping sickness). Sanofi stated that in clinical trials involving a total of 749 patients, fexinidazole demonstrated efficacy in treating both stages of sleeping sickness in children and adults. Dr. Nathalie Strub-Wourgaft, Director of Tropical Diseases at DNDi, said, “We look forward to fexinidazole becoming the first-line treatment for sleeping sickness, and we welcome the rapid approval of fexinidazole by the Democratic Republic of the Congo following the EMA’s approval, which demonstrates the commitment of the government of the Democratic Republic of the Congo, through its Ministry of Health, to eliminate sleeping sickness as a public health problem by 2020.”(SinaPharmaceuticals(Compiled by Fan Dongdong)

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1、Fexinidazole, the first all-oral treatment for sleeping sickness, approved in Democratic Republic of Congo

Source: Sina Medical News