Developing a comprehensive response for treatment of children under 6 years of age with schistosomiasis: research and development of a pediatric formulation of praziquantel.

Schistosomiasis is a parasitic disease caused by blood flukes. The disease is caused by an inflammatory reaction to parasite eggs retained in the liver, bladder and reproductive organs. According to 2017 World Health Organization (WHO) estimates 220 million people are potentially infected, from which probably 10% are children under 6 years of age. The regular treatment approach of a single, oral dose of 40 mg/kg body weight with praziquantel however, is difficult for children under the age of 6, leaving them without a treatment option. In order to address this important gap in treatment target populations, an international public-private partnership that works on a not-for-profit basis in the field of drug research and development for schistosomiasis was established in 2012. This is called the Pediatric Praziquantel Consortium. Its mission was and continues to be to develop, register and provide access to a suitable pediatric praziquantel formulation for treating schistosomiasis in preschool-age children (3-6 months up to 6 years). The Target Product Profile for the pediatric formulation of praziquantel that would be suitable to treat children as young as 3-6 months was then defined by a group of experts, including members from the Pediatric Praziquantel Consortium partner organizations as well as experts from WHO (as observer) and schistosomiasis endemic countries. The development of the drug is ongoing and the Pediatric Praziquantel Consortium aims to submit the regulatory dossier for marketing approval in endemic countries and WHO prequalification in 2018/19 with approval and product launch for schistosomiasis pediatric case management in key endemic countries in 2019. Ultimately, the goal is for the product to be considered for a large-scale mass distribution program by 2022.