Merck

• Arpraziquantel is a potential new treatment option for the estimated 50 million preschool-aged children with schistosomiasis, one of the most devastating parasitic diseases in the world
• A positive scientific opinion by EMA would facilitate registration of arpraziquantel in African endemic countries
• Merck is committed to the fight against schistosomiasis

Merck, a leading science and technology company, and the Pediatric Praziquantel Consortium, today announced that the 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 milestone, we are one step closer to achieving our mission of improving the health of preschool-aged children with schistosomiasis. Our contribution forms part of our larger ambition to eliminate schistosomiasis as a public health burden by 2030,” said Peter Guenter, Member of the Executive Board and CEO of Healthcare at Merck.

The application was submitted by Merck on behalf of the Consortium 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 scientific opinion by EMA, if received, will facilitate regulatory decisions in endemic countries. Merck is designated as the future Marketing Authorization Holder for African countries.

Derived from praziquantel, the standard of care treatment developed in the 1970s, arpraziquantel is tailored to meet the needs of preschool-aged children suffering from schistosomiasis. This group of approximately 50 million patients currently lacks a suitable treatment option. Containing the pharmacologically active enantiomer of praziquantel, arpraziquantel is a novel dispersible or orodispersible tablet (150mg). It can be taken with or without water, is palatable for preschool-aged children, and withstands hot and humid challenges presented by a tropical climate.

The clinical development program was completed at the end of 2021. In the 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.

For next steps, the Consortium is preparing for the potential inclusion of arpraziquantel in the World Health Organization list of prequalified and essential medicinal products. Together with relevant stakeholders, it is also exploring new mechanisms for providing equitable and sustainable access to arpraziquantel, once registered, and is conducting ADOPT – an implementation research program that paves the way for the large-scale delivery in endemic countries. The aim is to start the launch phases in 2024 for product availability on a not-for-profit basis in initial sub-Saharan African countries.

As part of the Merck’s Global Health strategic priorities, the company’s commitment to the schistosomiasis elimination and the related collaborative efforts, including the development of arpraziquantel, have been recognized in the 2022 Access to Medicine (ATM) Index where Merck ranks fifth among the 20 largest pharmaceutical companies based on initiatives to advance global access to medicines in low- and middle-income countries.

Merck aims to control and eliminate schistosomiasis through scientific and technological innovation. The strategy is not only to develop and provide medicines but also to improve diagnosis, counter disease transmission, increase disease control, expand access to healthcare, and strengthen local health systems. With this approach Merck is also helping to improve the health of underserved populations in low- and middle-income countries. By this the company contributes to United Nation’s Sustainable Development Goal 3 of good health and well-being.