European Medicines Agency recommends fexinidazole, the
first all-oral treatment for sleeping sickness
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The positive opinion is the result of a 10-year
partnership between the Drugs for Neglected Diseases initiative (DNDi), Sanofi and
African partners
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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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