The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) on Friday announced it has adopted a positive scientific opinion of Fexinidazole Winthrop (fexinidazole), the first oral-only medicine for the treatment of human African trypanosomiasis (HAT), commonly known as sleeping sickness.
This approval is a result of clinical trials led by the non-profit research and development organization DNDi, with an application submitted to EMA by Sanofi. CHMP’s decision means Sanofi can begin distributing fexinidazole in endemic countries in 2019, DNDi said.
HAT is a life-threatening, neglected tropical disease that is endemic in sub-Saharan Africa. There are two forms of sleeping sickness, depending on the parasite involved: Trypanosoma brucei gambiense
or Trypanosoma brucei rhodesiense
. The vast majority (98%) of reported cases are caused by T. b. gambiense
and most cases occur in the Democratic Republic of the Congo, according to EMA. The disease, transmitted by the bite of a tsetse fly, is usually fatal without treatment and about 65 million people are at risk, according to DNDi.
To develop fexinidazole, DNDi spent $62.5 million, which includes costs related to pre-clinical development and clinical studies, with the support of seven European countries (France, Germany, the Netherlands, Norway, Spain, Switzerland and the UK) as well as private donors including the Bill & Melinda Gates Foundation and Médecins Sans Frontières.
This is the 10th medicine recommended by EMA under Article 58, a mechanism that allows CHMP to assess and provide opinions on medicines for use outside the EU
. Medicines eligible for this procedure are used to prevent or treat diseases of major public-health interest and include vaccines and medicines for World Health Organization (WHO) target diseases such as HIV/AIDS, malaria or tuberculosis.
Medicines submitted under this program are assessed by EMA in collaboration with the WHO and must meet the same standards as medicines intended for EU citizens.