Case Study: Research and Development

Pediatric Praziquantel Consortium to Treat Schistosomiasis in Young Children, Astellas Pharma Inc.

Objectives

  • The Pediatric Praziquantel Consortium is a nonprofit partnership with Merck, TI Pharma, Swiss Tropical and Public Health Institute, Simcyp Ltd., and the Fiocruz Foundation, which combines the best science and most experienced public and private partners to develop, register, manufacture, and launch a new pediatric formulation of praziquantel, the current gold standard treatment for schistosomiasis. Our goal is to develop and register a new formulation of praziquantel that is suitable for healthcare professionals to administer to preschool-age children.
  • The consortium fully supports the World Health Organization (WHO) objective of eliminating schistosomiasis as a global health solution. This often neglected tropical disease affects more than 240 million people globally, including 100 million children.

Lessons Learned?

  • The consortium has overcome many scientific, regulatory, and access challenges in the area of global health through working as a team with our consortium partners and expert panel. We are actively focused on extending our partnership into endemic countries because this approach will allow us to encourage capacity building in those countries.
  • This partnership has evolved during the past two years. More partners have become involved, including from endemic countries; more funding has been made available, such as from the Bill & Melinda Gates Foundation and the Global Health Innovative Technology (GHIT) Fund; and each partners’ roles have expanded.

Results to Date?

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

  • A GMP (Good Manufacturing Practice) batch has been procured.
  • New oral dispersible pediatric PZQ formulation candidates have been developed. (See next bullet paragraph for details.)
  • A preclinical package to support the initiation of the phase I studies has been completed. So far, no toxicity issues have been reported.
  • The technological know-how behind the pediatric formulation process has been transferred to GMP manufacturers.
  • With the input of world-renowned clinical experts, a clinical development plan has been drafted and is being discussed with regulators.
  • Funding from the Bill & Melinda Gates Foundation and Global Health Innovative Technology (GHIT) Fund were secured to finance some important gaps identified in the preclinical and clinical programs.

Astellas pharmaceutical research and technology labs have developed a tablet that is a quarter the size of the current commercial praziquantel tablet (Cesol, which is 600 milligrams). Smaller tablets allows patients to take it without water and allows treatment (in principle) of very young children, including infants as young as three months.


More Information

www.astellas.com/jp/csr/pdf/health_01.pdf