Development and registration of a new praziquantel pediatric formulation for the treatment of schistosomiasis
Project Completed
 
  • Awarded Year
    2013
  • Awarded Amount
    $1,864,898
  • Disease
    NTDs Schistosomiasis
  • Intervention
    Drug
  • Development Stage
    Clinical Phase1
  • Collaboration Partners
    Astellas Pharma Inc., Farmanguinhos, Merck KGaA, Simcyp Limited, Swiss Tropical and Public Health Institute, Lygature

Introduction and Background of the Project

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 240 million people, including 100 million children, globally. It is the second most prevalent tropical disease 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 younger children (under 4 years) cannot be treated due to missing clinical data. 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 context of the WHO’s 2020 Roadmap and with respect to the initiative of “Uniting to combat Neglected Tropical Diseases” under which the global community agreed on control and elimination of schistosomiasis.

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. 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 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 2020 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 combines the best science and most experienced public and private partners to develop and register a new and robust pediatric formulation of praziquantel, the current gold standard treatment for schistosomiasis. It invests significantly in developing new formulation praziquantel candidates that are easy to administer to preschool-age children, infants and toddlers, exhibit improved taste, and are able to withstand the challenges presented by a tropical climate. Merck brings the necessary chemistry and manufacturing expertise and support related to praziquantel. It also provides the preclinical, clinical and regulatory resources necessary to efficiently and successfully execute the project. Astellas Pharma Inc. contributes its novel pharmaceutical technology which helps to improve drug compliance and functionality, reducing or eliminating the bitter taste of praziquantel. Swiss TPH brings extensive experience in schistosomiasis biological and pharmacological research, clinical research on drug effectiveness and efficiency in endemic regions and epidemiology. The governance is facilitated by TI Pharma, a 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. The Pediatric Praziquantel Consortium is currently concluding the pre-clinical phase and has met its objectives for that phase. The project is now moving into the clinical development phase.

Others (including references if necessary)

To overcome the many scientific, regulatory and access challenges it faces, the Pediatric Praziquantel Consortium continually seeks help from external experts and partners who wish to join. Beginning of 2014 Farmanguinhos and Simcyp have joined the Consortium. Farmanguinhos, the federal governmental pharmaceutical laboratory of the Fiocruz Foundation in Brazil, brings unique expertise to produce and distribute the new pediatric formulations product in endemic countries. Simcyp, a small UK research-based company, will build and validate a pharmacokinetic model that will allow a better prediction of the appropriate dosage for use in the pediatric clinical trials.