Millions of preschoolaged children affected by schistosomiasis are not receiving treatment due to the lack of a suitable medicine. To address this unmet need, the Pediatric Praziquantel Consortium has developed a potential new treatment option that has now been validated for review by the European Medicines Agency. This is a key milestone in the ongoing effort to eliminate this neglected tropical disease. As a member of the consortium, Swiss TPH conducted the clinical trials.
Today, the Pediatric Praziquantel Consortium announced that the European Medicines Agency (EMA) has validated for review the application for its potential new child-friendly drug for the treatment of schistosomiasis in preschool-aged children (3 months to 6 years of age). With this validation, the regulatory application for the pediatric drug is complete and EMA will now begin the scientific review process.
A child-friendly treatment tailored to the use in endemic countriesThe potential new pediatric treatment option is a novel dispersible or oro-dispersible tablet (150 mg). It can be taken with or without water, is palatable for preschool-aged children, and withstands the challenges presented by a tropical climate.
The Consortium, an international public-private partnership, successfully completed its clinical development program at the end of 2021. In its pivotal Phase III trial, the primary efficacy endpoint of clinical cure was met with a favorable safety profile. Adverse reactions observed in clinical studies were similar to those reported for praziquantel, the standard of care treatment in adults and school-aged children.
On behalf of the Consortium, Merck submitted the application for a scientific opinion by EMA under the EU-M4all procedure for high-priority medicines for human use intended for markets outside the European Union. If received, a positive opinion by EMA will facilitate the inclusion of the treatment in the World Health Organization list of prequalified and essential medicines, as well as regulatory decisions in endemic countries.
Swiss TPH conducted clinical trialsThe Swiss TPH is a founding member of the Pediatric Praziquantel Consortium and led the implementation of the clinical trials in endemic countries in sub-Saharan Africa from phase I to III. -We are proud to have contributed to the clinical development. The review by the EMA is a major milestone that brings us one step closer to the introduction of the potential pediatric treatment for the very young children in Africa,- said Eric Huber, Project Leader of the clinical trials at Swiss TPH.
With the regulatory filing stage complete, the Consortium is exploring new mechanisms for providing equitable and sustainable access to the drug, once approved. Through its dedicated access program, ADOPT, the Consortium is also paving the way for the large-scale delivery of the drug in endemic countries. The aim is to start the launch phase in 2024 for product availability on a not-for-profit basis in countries in sub-Saharan Africa. -We have come a long way and now have to go the last mile to get the treatment to the patients in need - the youngest children in countries affected by schistosomiasis,- said Peter Steinmann, public health specialist and coordinator of the ADOPT programme at Swiss TPH.