Aiming to Make Products Available to Children in Endemic Countries in Africa within the Next Few Years
Schistosomiasis- A Potentially Life-threatening Infection
Schistosomiasis is one of the most prevalent parasitic diseases in developing countries, notably in Africa and South America. The economic and health impacts of schistosomiasis are considerable. If it is left untreated, schistosomiasis causes anaemia, stunted growth, reduced ability to learn, and chronic inflammation of the organs, which can be fatal*1. The disease has a particularly high incidence among children.
The existing “standard of care” treatment for schistosomiasis is praziquantel. However, at present, the group of infected preschool-age children, including infants and toddlers, is left untreated in public health programs due to lack of relevant clinical data and a missing appropriate child-friendly formulation of the drug. Very young children cannot swallow the existing tablet because of its large size and bitter taste.
Pediatric Formulation Developed with Consortium Partners Based on Astellas’ Technology
Within a consortium of partners, involving pharmaceutical companies, research institutions, and international non-profit organizations, Astellas has developed a pediatric formulation of praziquantel.
The pediatric formulation newly developed by Astellas uses proprietary formulation technology to create a smaller, orally dispersible tablet of reduced bitterness that can be taken with or without water. The pediatric formulation has also been designed for production at a reduced cost using simple manufacturing processes, yielding tablets that are stable even in hot and humid tropical climates.
The pediatric formulation has been subsequently optimized by Merck in Germany, and technology and expertise transferred for clinical and manufacturing supply to Farmanguinhos in Brazil – both consortium partners. These activities have facilitated the production of materials for clinical trials and help develop local pharmaceutical manufacturing capabilities for future supplies.
Clinical Development of Pediatric Formulation
The clinical program was set up in line with European Medicines Agency (EMA) recommendations for pediatric development. It was designed with the support of regulatory authorities and a panel of international experts, including clinicians from endemic countries. It embedded a full phase I-III clinical development program. The pivotal phase III trial in Côte d’Ivoire and Kenya was completed in November 2021. The results of the trial confirm a favorable efficacy and safety profile for this potential new treatment option for pre-school aged children with schistosomiasis, in children 3 months to 6 years of age.
In December 2022, EMA has validated the regulatory application for the potential new treatment option for preschoolers and has started the review process.
In addition, in February 2021, the Consortium received additional funding from the Global Health Innovative Technology (GHIT) Fund and the European and Developing Countries Clinical Trials Partnership (EDCTP) to initiate implementation research programs to prepare for large-scale access and delivery of the potential novel pediatric treatment option in endemic countries. *2
In parallel, the Consortium is also exploring new mechanisms for equitable and sustainable access to the new medication, once it will be registered.
As a member of the Consortium, Astellas is committed to providing all necessary technical expertise and to continue working with the partners to have the product available for launch in the first endemic country in Africa within a few years.
For more information on the Consortium program and the partners, please refer to the following link: https://www.pediatricpraziquantelconsortium.org/
* Consortium activities are currently supported by grants from the Global Health Innovative Technology (GHIT) Fund and the European and Developing Countries Clinical Trials Partnership (EDCTP).
Newly developed pediatric formulation (top) and existing tablet (bottom)
Pediatric Praziquantel Consortium partners and project contributors
*This movie was created by GHIT Fund on July 2019.