Projects
Pediatric Praziquantel Consortium
Developing and providing a pediatric formulation of the drug praziquantel, to treat schistosomiasis in preschool-aged children
Latest project updates
- First Preschool-Aged Child Treated with the Pediatric Praziquantel Consortium’s New Pediatric Treatment for Schistosomiasis
- Technology transfer of new pediatric treatment option for schistosomiasis advances in Brazil
- New pediatric treatment option for preschoolers with schistosomiasis now included in WHO List of Prequalified Medicines
The Pediatric Praziquantel Consortium targets preschool-aged children with schistosomiasis, a parasitic disease that affects more than 240 million people worldwide. The consortium was established in July 2012 and uses an innovative R&D operating model that builds new partnerships as needed and shares development risks.
14
partners since 2021
12 years
duration of the program
About the Pediatric Praziquantel Consortium
Praziquantel (PZQ), the gold standard treatment for schistosomiasis, is currently available in large oral tablets that are suitable only for adults and school-aged children. Due to the size of the tablet, its bitter taste, and a lack of clinical data, an appropriate treatment for very young children does not exist. The consortium addresses this issue and has developed a new orally disintegrating PZQ formulation known as arpraziquantel. With the full clinical development phase successfully completed in 2021, the program has entered the regulatory filing stage, while preparing for the potential delivery of arpraziquantel through the consortium’s dedicated access program, ADOPT. The consortium is supported by grants from the Bill and Melinda Gates Foundation in 2013, the Global Health Initiative Technology Fund in 2013, 2014, 2016, 2019 & 2021, and the European & Developing Countries Clinical Trials Partnership (EDCTP) (2018 & 2021), under its second program supported by the European Union. Visit the Pediatric Praziquantel Consortium website for more information about the program.
Bridging industry interests and societal needs
Lygature helped to set up the consortium and fulfills an independent ‘broker’ role, a key ingredient of successful public-private development partnerships. Reflecting the consortium’s position at the crossroad between industry and society, the contributions being made by Lygature from January 2016 include:
- Coordination and governance. As consortium coordinator, Lygature provides necessary governance in terms of monitoring progress, finance, IP management, and collaborations. In addition, it successfully takes the lead in fundraising to support the Development and Access programs (current total value: EUR 5m).
- Communication and dissemination . Lygature pro-actively coordinates the engagement with key stakeholders to share the consortium’s ambition to make a meaningful contribution to tropical medicine and health innovation in endemic countries. Responsibilities include (but are not limited to): presenting at (inter)national conferences; writing of (scientific) articles and news items; and development of the consortium’s communication strategy, website, and visual identity and social media management.
The Pediatric Praziquantel Consortium is an example of the Lygature mission to bring together public and private partners, and so to make a meaningful contribution to global health. The project relies heavily on Lygature’s expertise in both management and science. In addition, Lygature has helped to ensure that each partner’s needs, interests, and objectives are met.
Lygature together with
Merck KGaA (Darmstadt, Germany), Astellas Pharma Inc. (Japan) , Swiss Tropical & Public Health Institute , Farmanguinhos, Unlimit Health, KEMRI, UFHB, Technischen Universität München, African Institute for Health and Development, Ministry of Health in Côte d’Ivoire, Ministry of Health in Kenya, Ministry of Health in Uganda, Makerere University School of Public Health
Project updates
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Pediatric Praziquantel Consortium presenting at the 67th ASTMH Annual Meeting
Latest clinical data will be presented by the Lead of the Consortium Pediatric Praziquantel program at the 67th ASTMH (American Society of Tropical Medicine & Hygiene) Annual Meeting on October 28-November 1, 2018 (New Orleans, US). In September, ASTMH informed the Pediatric Praziquantel Consortium that the abstract “Safety and efficacy of new oral dispersible tablet (ODT) formulations of racemate- and L-Praziquantel in 2 to 6 years old children infected with Schistosomiasis” was accepted for presentation.
ASTMH is the largest international scientific organization of experts dedicated to reduce the worldwide burden of tropical infectious diseases and improving global health. It will therefore be a great privilege for the Program Lead, Dr. Elly Kourany-Lefoll (from Merck KGaA, Darmstadt, Germany), to present the latest clinical data of the program at this important Annual Meeting designed for researchers, professors, government and public health officials, clinicians and healthcare professionals amongst others, that will host over thousands experts working in the field of global health. The presentation is planned on October 30th within the session “Schistosomiasis and Other Trematodes: Diagnosis and Treatment”.
In addition to this presentation, representatives from the Consortium partners will be available to respond to any specific questions on the Pediatric PZQ program at ASTMH as well as during the Global Schistosomiasis Alliance and COR-NTD meetings preceding the ASTMH conference. More on ASTMH is available at: www.astmh.org.
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Publication: Extrapolation of praziquantel pharmacokinetics to a pediatric population
In September 2018, members of the Pediatric Praziquantel Consortium published a paper on the extrapolation of praziquantel pharmacokinetics to a pediatric patient population in the peer-reviewed Journal of Pharmacokinetics and Pharmacodynamics.
The article discusses how caution is advised when extrapolating praziquantel pharmacokinetics from healthy adults to infected patients of 2-5 years of age. Important factors to consider in extrapolation models seem to be differences in meal composition between the standardized Phase I unit and clinical sites in Africa, and differences in study population composition.Many studies do not account for, or report on, the abovementioned differences, while this study shows they could greatly affect predicted exposure: simulated exposures were about tenfold lower compared to the clinical study results.
Links:
Publication in Journal of Pharmacokinetics and Pharmacodynamics: https://link.springer.com/article/10.1007/s10928-018-9601-1 -
Performance Materials at Merck KGaA, Darmstadt, Germany: Key Player towards the success of the Consortium Pediatric Praziquantel Program
The broad contribution by Merck KGaA, Darmstadt, Germany to the Pediatric Praziquantel Consortium includes the significant support by the Performance Materials business sector for the development of the PZQ Active Pharmaceutical Ingredient (API) and the timely material supplies for the clinical trials.