The Pediatric Praziquantel Consortium Announces Positive Phase III Results for Arpraziquantel To Treat Schistosomiasis
The Pediatric Praziquantel Consortium, a public-private partnership dedicated to the development of arpraziquantel, a potential new treatment option for schistosomiasis in preschool-aged children, announced the completion of its pivotal Phase III trial in Côte d'Ivoire and Kenya.
The results of the trial, co-funded by the Global Health Innovative Technology (GHIT) Fund and the European & Developing Countries Clinical Trials Partnership (EDCTP), confirm a favorable efficacy and safety profile for arpraziquantel in children 3 months to 6 years of age, affected by this neglected tropical disease.
Schistosomiasis is one of the most damaging parasitic diseases, affecting the lives of around 240 million people, and is highly prevalent in sub-Saharan Africa. The drug praziquantel – the current standard treatment developed in the 1970s – is safe, effective, and available for school-aged children and adults. At present, around 50 million preschool-aged children have been left untreated in public health programs primarily due to the lack of an appropriate child-friendly formulation of the drug.
Derived from praziquantel, arpraziquantel is an orally dispersible tablet (dissolves in the mouth). It was developed by Astellas Pharma Inc. in Japan, subsequently optimized by Merck in Germany and transferred for clinical manufacturing to Farmanguinhos in Brazil. The new tablet is small, has appropriate taste properties, can be taken with or without water, and withstands the hot and humid challenges presented by a tropical climate.
Dr Michael Makanga, Executive Director, EDCTP said: “With the completion of the Phase III trial, the Pediatric Praziquantel Consortium demonstrates that balanced North-South collaboration with complementary expertise, bidirectional knowledge sharing, and mutual trust, is a key success factor to develop and deliver safe and affordable treatments for neglected tropical diseases, such as schistosomiasis.”
With the full clinical development phase successfully completed, the program has entered the regulatory filing stage, while preparing for the potential delivery of arpraziquantel through the Consortium’s dedicated access program, ADOPT.
On behalf of the Consortium, Merck intends to apply 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. A positive opinion by EMA would facilitate the inclusion of arpraziquantel in the WHO list of prequalified medicinal products, as well as regulatory approvals in endemic countries.