Development of a new pediatric treatment option for schistosomiasis

EMA Recommends a new pediatric treatment option for Treatment of Schistosomiasis in Preschool-Aged Children

Main targets
Creating innovation

Schistosomiasis- A Potentially Life-threatening Infection


Schistosomiasis (also known as bilharzia)*1 is one of the parasitic diseases worldwide, spreading to 78 countries and endemic in 51 countries. More than 240 million people are affected, resulting in a significant 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 21 neglected tropical diseases (NTDs). If it is left untreated, schistosomiasis causes anemia, stunted growth, reduced ability to learn, and chronic inflammation of the organs, which can be fatal.

The current standard of care treatment for schistosomiasis is suitable for school-aged children and adults. However, 50 million preschool-aged children have not been treated in public healthcare programs mainly due to the lack of an appropriate formulation for the youngest.

Providing innovative formulation technologies and contributed to the development of a new pediatric treatment option as a member of 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 infected preschool-aged children. Its mission is to develop, register, and provide access to a suitable pediatric drug for treating schistosomiasis in children 3 months to 6 years of age.

Astellas has participated as a founding member of the Pediatric Praziquantel Consortium since 2012 and has been working with Consortium partners - pharmaceutical companies, research institutions, and international non-profit organizations - to (co)-develop a suitable pediatric formulation. 
Our innovative formulation technology played a pivotal role, especially in the development of initial formulations of the new pediatric treatment option.
The pediatric formulation is palatable for very young children by reducing the bitter taste associated with the medication and is administered via a 150mg dispersible tablet that is dissolved in water.

The prototype of the pediatric formulation has been developed by Astellas and further optimized by Merck (Germany) for global production using simple manufacturing processes yielding tablets that can remain shelf stable, even in hot and humid tropical climates.

Towards Access to a new pediatric treatment option for Endemic Countries in Africa

In December 2023, the European Medicines Agency (EMA) granted the positive Committee for Medicinal Products for Human Use (CHMP) scientific opinion on the new pediatric treatment option for preschool-aged children with schistosomiasis. Beyond the steps for inclusion of the new medication into the World Health Organization’s list of prequalified and essential medicinal products, the Consortium’s implementation research program (ADOPT)*2 is ongoing and preparing for the introduction of a new pediatric treatment option in the first endemic countries in Africa. To ensure equitable and sustainable access, it is essential that new procurement and funding mechanisms are collaboratively explored and established.

Astellas recognizes Access to Health as a materiality, and through our VISION to be "On the forefront of healthcare change to turn innovative science into VALUE for patients", we are proactively taking a comprehensive approach to solving this issue through the methods defined below.
(1) Astellas’ core business (Rx, Rx+)
(2) Enhancing availability of Astellas products (Access to Medicines) 
(3) Collaboration and support for the activities implemented by external partners

Initiatives in the Consortium apply to (3), and the positive CHMP (Committee for Medicinal Products for Human Use) scientific opinion by EMA of a new pediatric treatment option represents a significant milestone in alignment with Astellas’ Access to Health focus.

As a member of the Consortium, Astellas is committed to continue working with the partners to have a new pediatric treatment option available for children in schistosomiasis-endemic countries in Africa.

For more information on the Consortium's activities and partners, please see below.

  • Diseases of poverty, like schistosomiasis, must be overcome in order to deliver on the United Nations’ Sustainable Development Goals (SDGs) and ensure universal health coverage. The activity of the Consortium is making a tangible contribution to the elimination of schistosomiasis as a public health problem in particular SDGs 3 (Good Health and Wellbeing) & 17 (Partnerships for the Goals).
  • Consortium activities are currently supported by grants from the Global Health Innovative Technology (GHIT) Fund and the European and Developing Countries Clinical Trials Partnership (EDCTP).

*2 ADOPT: Adoption of Levo-Praziquantel 150 mg for schistosomiasis by endemic countries

Newly developed  pediatric formulation ©Merck

Newly developed  pediatric formulation



*This movie was created by GHIT Fund on July 2019.