European
Medicines Agency recommends fexinidazole, the first all-oral
treatment for sleeping sickness The positive opinion is the
result of a 10-year partnership between the Drugs for Neglected
Diseases initiative (DNDi), Sanofi and African partners
Fexinidazole will support international efforts to eliminate
sleeping sickness, a fatal neglected tropical disease endemic to
Africa Paris and Geneva - November 16, 2018 - The European
Medicines Agency's Committee for Medicinal Products for Human Use
(CHMP) has adopted a positive scientific opinion of fexinidazole,
the first all-oral treatment that has been shown 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. The
decision paves the way for the distribution of fexinidazole in
endemic countries in 2019. Sleeping sickness, or human African
trypanosomiasis (HAT), is usually fatal without treatment.
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. About 65 million people in sub-Saharan Africa are at
risk. "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
Kandé, who as 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 hospitalized 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." 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 (i) the early stage of the disease as well as the
(ii) second stage of the disease in which the parasites have
crossed the blood-brain 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 >= 6 years old and weighing
>= 20 kg. Results showed that fexinidazole could, therefore,
eliminate the need for systematic hospitalization 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."
Fexinidazole is a 5-nitroimidazole derivative that was rediscovered
in 2005, through collaboration with the Swiss Tropical and Public
Health Institute, during DNDi's search for compounds with
anti-parasitic activity, after being developed and then abandoned
for strategic reasons by Hoechst (now Sanofi) in the 1980s. In
2009, DNDi and Sanofi concluded a collaboration agreement for the
development, manufacturing, and distribution of fexinidazole, with
DNDi responsible for pre-clinical, clinical, and pharmaceutical
development, and Sanofi for industrial development, registration,
production, and distribution of the drug. "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 Organization as part of our mission to support the
elimination of sleeping sickness." In December 2017, Sanofi
submitted a regulatory dossier to the European Medicines Agency
under Article 58 of Regulation 726/2004, an innovative regulatory
mechanism intended for the review of new medicines destined for use
outside of the European Union. By allowing for the participation of
endemic countries (DRC and Uganda) and of the WHO in the evaluation
of the fexinidazole regulatory dossier, approval under Article 58
also facilitates and could accelerate future national product
registrations and patient access. "Together with Ministries of
Health in endemic countries we have shown it is possible to conduct
high quality trials in the most challenging settings," said Dr.
Nathalie Strub-Wourgaft, DNDi Director of Neglected Tropical
Diseases. "This is only the first step - we now need to ensure
patients can access and benefit from this new drug." To develop
fexinidazole, DNDi spent EUR 55 million (USD 62.5 million), which
includes costs related to pre-clinical development and clinical
studies. The project was supported by 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. About sleeping
sickness The majority of sleeping sickness patients are
reported in the Democratic Republic of Congo, where 78% of
Trypanosoma brucei gambiense sleeping sickness cases were reported
in 2017, followed by the Central African Republic, Guinea and Chad.
The latest data released by the WHO in July 2018 confirm a
sustained decrease in the number of new cases. Only 1,447 new cases
were reported to the WHO in 2017 compared to 2,164 cases in 2016
and 9,870 cases in 2009. But the history of sleeping sickness is
marked by resurgence, interspersed by decades where the disease has
seemed largely under control. In its roadmap on neglected tropical
diseases published in 2012 and supported the same year by the
London Declaration, the WHO identified sleeping sickness as a
public health problem, and targets its elimination by 2020.
About DNDi A not-for-profit research and development
organization, DNDi works to deliver new treatments for
neglected diseases, in particular human African trypanosomiasis,
leishmaniasis, Chagas disease, filarial infections, mycetoma,
paediatric HIV, and hepatitis C. NECT is one of the seven
treatments delivered by DNDi since its inception in 2003.
Fexinidazole is the first new chemical entity to be successfully
developed by DNDi. DNDi's fexinidazole programme is supported by
grants from the Bill & Melinda Gates Foundation, USA; UK aid,
UK; Dutch Ministry of Foreign Affairs (DGIS), The Netherlands;
Federal Ministry of Education and Research (BMBF) through KfW,
Germany; French Development Agency (AFD), France; German
International Cooperation (GIZ), Germany; Ministry of European and
Foreign Affairs (MEAE), France, Médecins sans Frontières; Norwegian
Agency for Development Cooperation (Norad), Norway; Republic and
Canton of Geneva, Internal Solidarity Office, Switzerland; Spanish
Agency for International Development and Cooperation (AECID),
Spain; Swiss Agency for Development and Cooperation (SDC),
Switzerland; UBS Optimus Foundation, Switzerland; Brian Mercer
Charitable Trust, UK; Stavros Niarchos Foundation, USA and other
private foundations and individuals from the HAT campaign. |
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