Introduction and Background of the Project

Introduction

Schistosomiasis, also known as bilharzia and endemic in 78 developing countries, is a chronic inflammatory neglected tropical disease caused by parasitic worms. The disease affects more than 206 million people, including 100 million children, globally. It is one of the most prevalent tropical diseases in the world after malaria, and represents an important health burden in developing countries, especially in Africa where more than 90% of the infections occur. 

The current gold standard recommended treatment for schistosomiasis, praziquantel (PZQ), is available in oral tablets for adults and children, but the effective dose for children < 4 years is currently not known as pharmacokinetics or dose-finding clinical studies have never been systematically conducted in this age group until recently. In addition, a pediatric formulation of praziquantel that would be appropriate for preschool age children, infants and toddlers, and would permit accurate dosing and enhanced compliance in these patients is non-existent and highly needed. The development of a new pediatric formulation of praziquantel is to be seen in the current context of the WHO’s 2020 Roadmap and with respect to the upcoming NTD Roadmap 2021-2030 under which the global community agreed on control and elimination of schistosomiasis.

Project objective

The Pediatric Praziquantel Consortium is an international not-for-profit partnership that aims to reduce the global disease burden of schistosomiasis by addressing the medical need of infected preschool-age children. Its mission is to develop, register and provide access to a suitable pediatric praziquantel formulation for treating schistosomiasis in this age group. The pediatric formulation, currently under investigation in a Phase III clinical trial, has been designed to be smaller, exhibit an improved palatability and be orally dispersible compared to the current commercial formulation. The next steps will focus on establishing optimal Levo-Praziquantel access and delivery strategies for different endemic settings in Africa.

Project design

The Consortium was established in July 2012 by Merck KGaA, Darmstadt, Germany (Merck KGaA), Astellas Pharma Inc. (Astellas), Swiss Tropical and Public Health Institute (Swiss TPH) and Lygature (formerly TI Pharma). At the beginning of 2014, Farmanguinhos and Simcyp, a Certara company, joined the Consortium as full partners. The Schistosomiasis Control Initiative (SCI) joined in 2016. At the end of 2017 Simcyp completed all its tasks around PK modeling and left the Consortium. In Jan 2018 Université Félix Houphouët Boigny (UFHB) and Kenya Medical Research Institute (KEMRI) joined the Consortium as new partners. In 2020, the Consortium will be strengthened with the Technical University of Munich (TUM). Since the start, all partners have been efficiently working together to progress the project and achieve the pre-clinical, clinical phase I-III and registration objectives.

All partners are bound by a Consortium agreement, which arranges their roles and responsibilities and includes a formal governance structure with a Consortium Board as the highest decision-making body, and Lygature as independent Coordinator. An external expert panel is called by the Consortium prior to each clinical development stage to provide an independent evaluation of the project. The partners have formed a core project team, led by Merck KGaA, comprising one representative of each partner and/or expertise area that meets every month. The core project team is supported by various subteams focusing on specific technical and operational aspects to implement the consortium’s program.

How can your partnership (project) address global health challenges?

In order to tackle the important public health problem of schistosomiasis, the non-profit Pediatric Praziquantel Consortium has been formed in July 2012 to develop a pediatric formulation of praziquantel. The project will contribute to the overall goal of control and elimination of schistosomiasis by developing a product urgently required for the treatment of preschool-age children, infants and toddlers, a population that is currently being considered by WHO to be included in treatment campaigns. The development and registration of a new pediatric formulation of praziquantel is regarded by the Consortium as a cornerstone to fulfill the WHO commitment to address the health burden that schistosomiasis represents and the importance of controlling disease-related morbidities. To achieve this commitment, several measures need to be integrated among which making chemotherapy treatment available to all age groups is an important part of.

What sort of innovation are you bringing in your project?

The Pediatric Praziquantel Consortium has developed and optimized pediatric orodispersible tablet (ODT) formulations of the enantiopure R-(-)-PZQ and racemate PZQ (back up) that will permit preschool-age children to be targeted for treatment thus closing the major treatment gap mentioned above. The target was to have a new ODT formulation that preferably contains the enantiopure R-(-)-praziquantel (L-PZQ ODT), since the schistosomicidal activity arises only from R-(-)-praziquantel, which has also been reported to be less bitter than racemate PZQ. Based on the outcome of the Phase I program and discussions with key stakeholders, including WHO PQP and WHO NTD, the Consortium included both formulations in the Phase II trial in children (age 3 months to 6 years) infected with S. mansoni in Cote d’Ivoire. Data from the Phase II pediatric trial (2-6 years) led to the selection of the L-PZQ ODT formulation at a dose of 50 mg/kg to be pursued for the phase III trial, the current stage of the development program.

Role and Responsibility of Each Partner

Merck KGaA (Darmstadt, Germany) leads the program and brings expertise and support related to praziquantel, including resources from different areas (drug product manufacturing, pre-clinical, clinical, regulatory) needed for clinical development & registration and to efficiently execute the project. Astellas contributes by providing in-kind expert advice to support communication activities of the Consortium in order to secure procurement and funding for large-scale distribution of Levo-Praziquantel across endemic countries.. Swiss TPH brings extensive experience in helminths biological and pharmacological research, epidemiology and clinical research in endemic regions. The governance is facilitated by Lygature, a Dutch non-profit organization and independent party with an extensive portfolio of international public-private partnerships in drug research and development, including in the area of neglected diseases. KEMRI and UFHB will provide expertise on local disease epidemiology and clinical care, considered key elements of the access strategy. SCI provides the necessary expertise to define and execute the access strategy and plan as well as to initiate and implement NTD control and elimination programs in Sub-Saharan Africa. Finally, TUM, represented by the Center for Global Health, complements the Consortium with its multidisciplinary and multicultural expertise in the field of neglected tropical diseases with a special focus on social science in global health.

Others (including references if necessary)

For more detailed information on the Pediatric Praziquantel Consortium, the project team and the development program, please feel free to visit the Consortium website:

www.pediatricpraziquantelconsortium.org

To receive regular updates on the progress and activities of the program, feel free to sign-up for the bi-annual Consortium Newsletter:

http://www.pediatricpraziquantelconsortium.org/nieuwsbrief.html

Final Report

1. Project objective

The Pediatric Praziquantel Consortium is a non-profit international partnership committed to reducing schistosomiasis in preschool-age children (3 months to 6 years) by developing and delivering child-friendly medication. Since 2012, partners have progressed through pre-clinical and Phase I–III trials. Project G2020-102 focused on preparing this pediatric treatment for implementation by pursuing the following objectives: 

1. 1. To identify enablers and barriers to implementation, and define a social mobilization, communication and training strategy; 

1. 2. To assess existing and select optimal access platforms and delivery approaches;  

1. 3. To model future drug and funding needs for upscaling treatment implementation in routine schistosomiasis control programs. 

2. Project design

Uganda was selected as the study country. The Consortium operates through a transparent, agile governance model coordinated by Lygature (Netherlands). Technical University of Munich (Germany) led research on knowledge, acceptability, and perception to understand community needs. Unlimit Health (UK) drove advocacy and social mobilization to strengthen engagement. Swiss TPH (Switzerland) shaped delivery strategies, including alternatives to weight-based dosing. Merck (Germany) supplied the product while advancing technology transfer for local manufacturing, procurement planning, and launch preparedness—covering product readiness, access pathways, and national preparedness for implementation. Pilot implementation was through Ugandan partners, mainly the national schistosomiasis control program and Makerere University.

3. Results, lessons learned

Over four years, the Consortium achieved key milestones and advanced protocols for treating schistosomiasis in preschool-age children with the new pediatric medication. Key regulatory achievements included European Medicines Agency’s positive scientific opinion and submission to Uganda Health Authorities for national approval which was granted in August 2025 Inclusions on WHO Prequalification list (April 2024) and WHO’s Essential Medicines Lists for adults and children (July 2025) were achieved. These steps confirmed product quality, safety, and efficacy.

A historic result was the first child treated in Uganda in 2025, signaling the start of real-world use.

Pilot implementation generated strong evidence on feasibility, safety and acceptability. High treatment coverage in both distribution platforms — 89% (Neglected Tropical Diseases - Mass Drug Administration) and 79% (Child Health Days) in Uganda, demonstrated that the new treatment can be successfully integrated into existing distribution platforms. No serious adverse events were detected, and caregivers as well as health workers consistently reported high acceptance. A study of alternative weight estimation approaches identified the PAWPER XL-MAC tape as a promising alternative to scale-based dosing. 

Country readiness for routine adoption also progressed. National registration processes were initiated in early-adopter countries, training materials were updated, and implementation research protocols were developed. Social science research improved understanding of caregiver expectations, health-worker needs, communication gaps and structural barriers to treatment uptake, all of which were incorporated into the final tools and materials.

Manufacturing and supply-chain preparedness advanced substantially. Nearly 900,000 tablets were supplied for pilot studies, and sufficient active pharmaceutical ingredient (API) was secured to produce up to 5 million tablets. Engagement with international organizations supported exploration of pooled procurement mechanisms to optimize country access. 

An implementation toolkit is being developed covering planning, stakeholder engagement, advocacy and social mobilization, training and monitoring. Lessons learned emphasized early alignment between clinical, regulatory, and access teams; embedding social science in product introduction planning; and adopting flexible delivery models informed by systematic assessment of country health systems. Strong governance and long-term partnerships were critical to navigating complex public-private collaborations.