TOKYO, March 4,
2025 /PRNewswire/ -- The Global Health
Innovative Technology (GHIT) Fund is today announcing a significant
milestone: the first administration of arpraziquantel to
preschool-aged children in Uganda,
in an implementation science setting. This new pediatric treatment
for schistosomiasis, developed by the GHIT-funded Pediatric
Praziquantel Consortium, is being introduced through the
Consortium's ADOPT program, which focuses on integrating
arpraziquantel into existing healthcare platforms in countries
where schistosomiasis is prevalent. This achievement marks GHIT's
first supported innovation to reach people in need since its
establishment in 2013.
"This isn't just a scientific achievement," said Osamu Kunii, CEO of the GHIT Fund. "It's a
testament to the transformative power of global collaboration. By
bringing together a diverse array of partners, each steadfastly
contributing their unique expertise and sustaining momentum
throughout a decade-long marathon of innovation to shepherd this
breakthrough (from bench to bedside), arpraziquantel's development
journey has unlocked solutions that once seemed impossible."
Schistosomiasis, or bilharzia, affects 250 million people
globally, including an estimated 50 million preschool-aged
children, mainly in sub-Saharan Africa. Left untreated, this
poverty-related parasitic disease can lead to anemia, stunted
growth, and impaired learning ability, as well as chronic
inflammation of the organs, which can be fatal. Until now, a
child-friendly treatment specifically tailored for preschool-aged
children was not available, leaving millions of preschoolers at
risk.
Early treatment of schistosomiasis in preschool-aged children is
a crucial investment in public health and societal wellbeing. By
intervening at this critical stage, we not only aim to shield young
bodies from severe complications like organ damage and cognitive
impairment but also alleviate the long-term burden on healthcare
systems and hopefully better livelihoods.
The Pediatric Praziquantel Consortium developed arpraziquantel
to address this gap. Japan's
Astellas Pharma Inc., as a founding member of the Pediatric
Praziquantel Consortium, played a pivotal role by utilizing its
proprietary technology to lead arpraziquantel's initial formulation
development, resulting in water dispersible, climate-stable,
child-friendly tablets with acceptable taste. The formulation was
optimized by Merck in Germany; the
manufacturing process served to produce clinical trial supply from
Merck and Farmanguinhos in Brazil.
Current manufacturing is done by Farmanguinhos; future large-scale
production by Universal Corporation Ltd. in Kenya is planned in and for Africa.
"Japanese innovation is a cornerstone of arpraziquantel's
development," Dr. Kunii added, "embodying GHIT Fund's core mission:
harnessing the power of international partnerships to revolutionize
global health."
GHIT Fund has supported arpraziquantel's development journey
throughout the overall development program spanning over 10 years.
GHIT has and continues to support the Pediatric Praziquantel
Consortium in developing their access plans, with a particular
focus on exploring new and sustainable access mechanisms. GHIT is
also co-funding, with EDCTP, implementation via the Consortium's
ADOPT Program, which is assessing different implementation
platforms in three countries: Côte d'Ivoire, Uganda and Kenya. In addition, GHIT is coordinating,
together with the consortium, in-country readiness preparations for
introduction of arpraziquantel in additional countries such as
Senegal and Tanzania.
"Understanding what needs to be in place at country level in
terms of policy framework, regulatory framework, implementation
models and domestic resource mobilization is critical to the
successful uptake and roll out of arpraziquantel. We hope the
lessons learnt from these countries will inform other countries,"
said Dr Isaac Chikwanha, Senior
Director of Access at the GHIT Fund.
Arpraziquantel received European Medicines Agency's positive
scientific opinion in December 2023
and was included into the World Health Organization (WHO)'s List of
Prequalified Medicines in May 2024.
Inclusion in the WHO's Essential Medicines List is expected in
2025.
1. About schistosomiasis
Schistosomiasis (also
known as bilharzia) is one of the most prevalent parasitic diseases
worldwide and a very important one in terms of public health burden
and economic impact. It is a poverty-related disease that is
widespread in tropical and subtropical regions where large sections
of the population have no access to clean water. Flatworms transmit
the disease and people become infected with the parasite through
contact with freshwater, for example, while working, swimming,
fishing, or washing their clothes. The minuscule larvae penetrate
human skin, enter the blood vessels, and attack internal organs.
The infection rate is particularly high among children.
Schistosomiasis is a chronic condition and is classified by the
World Health Organization (WHO) as one of the 21 neglected tropical
diseases (NTDs).
https://www.who.int/en/news-room/fact-sheets/detail/schistosomiasis
2. About arpraziquantel
The current standard of
care treatment for schistosomiasis is praziquantel. Praziquantel is
already approved, and suitable for school-aged children and adults.
Extending the range of options for the treatment of
schistosomiasis, arpraziquantel is tailored for preschool-aged
children against Schistosoma mansoni and Schistosoma
haematobium. Tested in clinical development, under the
responsibility of Merck, arpraziquantel is a 150mg dispersible
tablet. The prototype of its pediatric formulation was developed by
Astellas Pharma Inc. in Japan, and
further optimized by Merck in Germany.
In developing arpraziquantel, the Pediatric Praziquantel
Consortium established a pediatric drug development program,
divided into four major steps: preclinical development, clinical
development, registration, and access. All details can be found on
the Consortium website.
3. About the Pediatric Praziquantel
Consortium
The Pediatric Praziquantel Consortium is an
international public-private partnership that aims to reduce the
global disease burden of schistosomiasis and improve child health
by addressing the medical needs of preschool-aged children. Its
mission has been to develop, register, and provide access to a
suitable pediatric drug for treating schistosomiasis in children 3
months to 6 years of age. For more information, and to see an
overview of all Consortium partners, visit the Consortium
website:
www.pediatricpraziquantelconsortium.org
The GHIT Fund is a Japan-based
international public-private partnership (PPP) fund that was formed
between the Government of Japan,
multiple pharmaceutical companies, the Bill & Melinda Gates
Foundation, Wellcome, and the United Nations Development Programme
(UNDP). The GHIT Fund invests in and manages an R&D portfolio
of development partnerships aimed at addressing neglected diseases,
such as malaria, tuberculosis, and neglected tropical diseases,
which afflict the world's vulnerable and underserved populations.
In collaboration with global partners, the GHIT Fund mobilizes
Japanese industry, academia, and research institutes to create new
drugs, vaccines, and diagnostics for malaria, tuberculosis, and
neglected tropical diseases.
https://www.ghitfund.org/en
For more information, contact:
Katy Lenard at +1-301-280-5719 or
klenard@burness.com
Eriko Mugitani at +81-36441-2032 or
eriko.mugitani@ghitfund.org
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SOURCE GHIT Fund
