First exclusively oral treatment for sleeping sickness is approved

Publicação: 12 de December de 2018

The World Health Organization considers the disease a public health problem and sets the goal to eliminate it by 2020. The new treatment renews hopes that it can be completely eliminated

The new treatment may improve the lives of thousands of patients in West and Central Africa, where the disease causes severe disturbances in sleep patterns, aggression, psychosis and death

The first exclusively oral treatment (fexinidazole) for sleeping sickness, or human African trypanosomiasis (TAH), received in November, a stamp of approval by the Committee for Human Medicinal Products (CHMP) of the European Medicines Agency (EMA). The recommendation is the result of clinical trials led by the Drugs for Neglected Diseases Initiative (DNDi) – a non-profit research and development organization – and a registry application submitted by Sanofi-Aventis. The decision opens the paths for individual countries to approve its use, which should happen within 90 days.

Fexinidazole was assessed in three clinical studies with 749 patients in different stages of the disease. Studies have shown high cure rates after ten days of treatment, especially in the early stages. The drug is intended to be marketed exclusively outside the European Union and, according to DNDi, patients should receive the medicine until mid-2019.

DNDi’s Director of Neglected Tropical Diseases, Nathalie Strub-Wourgaft, points out that this is great news on many levels. According to her, the disease can be cured faster since patients won’t need to travel for treatment, not only benefiting them, but also helping to slow the spread of the disease.

Current treatments

Current treatments include intramuscular injections of pentamidine, which are only effective in the early stages of the disease. Other treatments include medications eflornithine and nifurtimox, which demand a complicated series of infusions, requiring patients to undergo painful lumbar punctures to verify if the parasite is present in the spinal fluid, besides pills that this treatment must be held in hospital environments. In other words, they require a minimal health infrastructure and personnel, which are not promptly available in some remote areas.

Treatment with fexinidazole

The new drug is the first treatment exclusively conducted with oral medications that is effective both in the first and second stages of the disease, in which the parasites cross the patient’s blood-brain barrier. With this medication, healthcare professionals no longer need to test the spinal fluid. In addition, patients do not need to be hospitalized, reducing the demand for hospital beds and staff in poor countries.

Fexinidazole was originally developed in the late 1980 and abandoned by Hoechst, a German company that owned it. In 2005 it was rediscovered by DNDi researchers who were looking for possible antiparasitic compounds. DNDi cooperated with pharmaceutical company Sanofi to conduct the human trials and request an EMA recommendation under a special set of rules built to help low and middle-income countries to access medications outside the European Union.

Dr. Bernard Pécoul, Executive Director of DNDi, explains that the fexinidazole is a completely new compound developed through an alternative nonprofit Research & Development model. It’s the first new chemical entity developed by DNDi. Still according to him, this great therapeutic milestone highlights the unique partnership that DNDI has with Sanofi to discover, develop and record treatments for an extremely neglected illness.

The disease

Human African trypanosomiasis is caused by the parasite Trypanosoma brucei gambiense. It is transmitted by the bite of the tsetse fly and is usually lethal if left untreated. The disease causes neuropsychiatric symptoms including aggression, psychosis and a debilitating disorder of the sleep patterns. About 65 million people living in sub-Saharan Africa are at risk of being infected.

The majority of registered patients live in the Democratic Republic of Congo, where 78% of the cases were reported in 2017, followed by the Central African Republic, Guinea and Chad. The latest data published by the World Health Organization (WHO), in July 2018, confirm a steady decline in the number of new cases, with only 1,447 reported in 2017. In 2016, there were 2,164 cases and in 2009, the number reached 9,870 cases.

“As a physician, I dedicated my career to sleeping sickness. An exclusively oral treatment has been my dream for decades. The affected populations are among the most vulnerable and live in some of the most remote areas of Congo, if not the world. They need a safe, simple and effective treatment”, says Dr. Victor Kande, a neglected tropical diseases expert consultant of the Ministry of Health of the Democratic Republic of the Congo, who was chief investigator during the clinical trials. “Less than ten years ago we still treated this disease with an arsenic derivative that killed 5% of patients. The fexinidazole is distributed in the form of a simple pill: this is a huge leap in fighting this deadly disease”, he says.

To Michel Lotrowska, President of DNDi Latin America, “this story of success brings new perspectives to the development of medicines against neglected diseases and shows that an alternative model that does not target profit and that is focused on the needs of patients is possible”. According to him, “Latin America could eliminate some diseases that reach underserved populations if there was a joint effort of the countries of the region”.

Watch the story of how the fexinidazole, the first oral drug for sleeping sickness, was developed: the doctor’s dream: A pill for sleeping sickness.

Meet the researchers and doctors who believed their patients deserved the best medicine. Learn more by clicking here.