Pediatric Praziquantel PZQ Consortium

Partnership objective

Develop a new pediatric Praziquantel (PZQ) formulation to treat young children (under 6 years of age) for schistosomiasis.

Two novel PZQ formulation candidates have been developed by the Consortium. Two novel PZQ formulation candidates have been developed by the Consortium. Copyright Lygature

What are the health needs and challenges?

Schistosomiasis is one of the most prevalent parasitic diseases in Africa, and a very important one in terms of public health burden and economic impact. Left untreated, this poverty-related disease can lead to anemia, stunted growth and impaired learning ability, and chronic inflammation of the organs, which can be fatal in the most serious cases.

As efforts focus on morbidity control and elimination, there is a pressing need to treat preschool children (under 6 years old). No suitable drug formulation is made available for this high-risk group, which accounts for about 10% of the 220 million people already infected.

The existing ‘gold standard’ treatment for schistosomiasis is praziquantel (PZQ), which was developed in the 1970s. This oral anthelmintic is available as generic drug and currently donated through the Merck Praziquantel Donation Program, via the World Health Organization (WHO), to fight schistosomiasis in Africa. It is safe and effective, and a tablet formulation is available for adults and school-age children but not for those below 6 years of age.

Description of partnership activities and how they address needs and challenges

The Pediatric Praziquantel Consortium was founded in July 2012 as the first international, non-profit, public-private partnership in schistosomiasis, supported by world leading experts in tropical parasitic infectious diseases. The Consortium aims to develop, register and provide access to a pediatric praziquantel formulation for treating schistosomiasis in preschool-age children, with proven efficacy and safety and acceptable taste properties. The formulation should also withstand the challenges presented by a tropical climate.

Two novel PZQ formulation candidates have been developed by the Consortium: a racemic mixture and an enantiopure version, both with improved taste properties.

The new formulation candidates are a quarter of the size of the current approved PZQ Cesol® 600 mg tablet. Because they are orodispersible, they can be dispersed in water and administered to younger children, infants and toddlers. Both formulation candidates are currently in clinical Phase II testing.

The Consortium fully supports the WHO objective of eliminating schistosomiasis as a global health problem and is building a sustainable partnership through working with credible and experienced partners:

Merck (Germany) leads the program and provides expertise and support relating to PZQ, including internal resources from different areas needed for clinical development: drug product manufacturing; preclinical; clinical and regulatory. The Consortium is governed by a Board which consists of top management representatives from each partner.

Astellas Pharma Inc. (Japan) has developed the new pediatric PZQ formulations, and provides expert advice on clinical development in children, and pharmacokinetic modeling.

Lygature, a Dutch not-for-profit foundation, acts as the independent coordinator of the Consortium, providing governance in terms of progress, finance and collaboration. Since 2006, Lygature has supported close to a hundred public-private partnerships in the field of life sciences & health, including poverty-related diseases.

Swiss Tropical & Public Health Institute is a not-for-profit Institute internationally renowned for its research, services, teaching and training in global heath. It contributes with extensive experience in helminths biological and pharmacological research; epidemiology; and clinical research in endemic regions.

Farmanguinhos, the federal governmental pharmaceutical laboratory of the Fiocruz Foundation in Brazil, brings unique expertise to addressing the production and distribution of new pediatric formulations in endemic countries

SimCYP, a UK-based research company, provides pharmacokinetics modeling capabilities and expertise.

The Schistosomiasis Control Initiative (SCI) at Imperial College London, with a 15-year-experience in providing treatments against schistosomiasis and three soil-transmitted helminths to the rural poor in Sub-Saharan Africa and Yemen, facilitates preparation and implementation of the Access and Delivery plan in schistosomiasis endemic countries, to ensure optimal use for the new pediatric praziquantel formulation.

In addition, apart from in-kind contributions by the partners and a cash contribution by Merck, the Consortium has been supported by grants from the Bill and Melinda Gates Foundation awarded in 2013, and from the Global Health Innovative Technology (GHIT) Fund, awarded in 2014, 2015 and 2016.

Lessons learned      

The Consortium has addressed many scientific, regulatory and access challenges in the area of global health through working as a team with all consortium partners and involving pharmaceutical, (non-)clinical, clinical and regulatory experts in the field of pediatric medicine and schistosomiasis at key stages of the development process. The Consortium also actively explores opportunities to extend the partnership into endemic countries (and the partnership with Farmanguinhos in Brazil is an example) as it will contribute to building capacity in those countries.

Summary of impact and forward looking information

Since its implementation in July 2012, the consortium has accomplished the following: 

  • Preclinical package completed with no toxicity issues reported.
  • Process development and manufacturing of GMP material for Phase I and II completed.
  • ODT (orally disintegrating tablet) formulations developed and optimized.
  • Tech Transfers of the pediatric formulation processes, involving Consortium partners (Astellas, Merck and Farmanguinhos) to produce GMP batches for the clinical supplies, were completed.
  • Target Product Profile (TPP), clinical development program and initial regulatory strategy defined together with experts from endemic countries. 
  • Meetings with regulators held. 
  • TPP reviewed at the WHO NTD Informal consultation meeting on treatment of Schistosomiasis in preschool-age children and pediatric Praziquantel formulations (September 2015), resulting in only minor amendments.
  • Two Phase I bioavailability studies in healthy volunteers (South Africa) completed; the two new pediatric formulations well tolerated.
  • A swill-and spit taste study with school-age children (Tanzania) completed; both formulations were significantly more palatable than the commercial PZQ tablets.
  • Phase II study in S.Mansoni (schistosomiasis)-infected pre-school children implemented in Ivory Coast to assess the efficacy and safety of two orodispersible tablet formulations.
  • Grants from two international global health funds awarded to the Consortium to finance the program 

Partnership information

Company(ies) Astellas , Merck

Partner(s) Farmanguinhos, Lygature, Schistosomiasis Control Initiative, SimCyp, Swiss Tropical and Public Health Institute (Swiss TPH)

Type of Partner(s) Academia / Hospitals, Government, NGOs, Other Business

Therapeutic Focus Neglected Tropical Diseases

Disease(s) Schistosomiasis

Program Type(s) Availability of Treatment - Differential Pricing, Availability of Treatment - Technology Transfer - Scientific Collaboration and Knowledge Sharing, Prevention Programs - Mass Drug Administrations (MDA), Research & Development - Development of Treatments, Research & Development - Pediatric R&D

Targeted Population(s) Children, People with low income

Region(s) World

Number of Countries 1

Country(ies) Worldwide

Start Date 2012

More information Eliminating schistosomiasis in Young Children

Anticipated completion date Ongoing

« By entering the phase of clinical testing in infected young children in Africa, the program has achieved an important milestone towards bringing an innovative product for NTDs to the patients in need. »

J. Reinhard-Rupp, Head of Global Health, Merck