European Medicines Agency Validates Application for Arpraziquantel
The Pediatric Praziquantel Consortium announced on 2nd December 2022 that the European Medicines Agency (EMA) has validated for review the application for arpraziquantel for the treatment of schistosomiasis in preschool-aged children (3 months to 6 years of age). With this validation, the regulatory application for arpraziquantel is complete and EMA will now begin the scientific review process.
Derived from praziquantel, the standard of care treatment developed in the 1970s, arpraziquantel is tailored to meet the needs of preschool-aged children affected by schistosomiasis. This group of approximately 50 million patients currently lacks a suitable treatment option. Arpraziquantel is a novel dispersible or orodispersible tablet (150 mg). It can be taken with or without water, is palatable for young children, and withstands the hot and humid 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.
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 regulatory decisions in endemic countries. Merck is designated as the future Marketing Authorization Holder for African countries.
“Having reached this milestone after 10 years of intense and collaborative work makes me very proud. We are now close to our common goal of improving children’s health with a potential innovative and suitable treatment option for the very young patients suffering from schistosomiasis,”
Jutta Reinhard-Rupp
Chair of the Pediatric Praziquantel Consortium Board and Head of the Global Health Institute at Merck www.pediatricpraziquantelconsortium.org
With the regulatory filing stage complete, the Consortium is preparing for the potential inclusion of arpraziquantel in the World Health Organization list of prequalified and essential medicines. It is exploring new mechanisms for providing equitable and sustainable access to arpraziquantel, once approved. Through its dedicated access program, ADOPT, the Consortium is also paving the way for the large-scale delivery of the potential new treatment option in endemic countries. The aim is to start the launch phase in 2024 for product availability on a not-for-profit basis in initial sub-Saharan African countries. At the moment, arpraziquantel is not approved for use in any country.