Schistosomiasis, a debilitating parasitic disease also known as bilharzia or snail fever, affects an estimated 250 million people globally, with a disproportionate impact on preschool-aged children in sub-Saharan Africa. This neglected tropical disease, caused by parasitic worms residing in freshwater snails, leads to a cascade of health complications, including malnutrition, anemia, stunted growth, and cognitive impairment. Chronic infections can result in organ damage and even death. Until recently, the absence of a child-friendly treatment option left millions of young children vulnerable to the devastating effects of this preventable disease. The Pediatric Praziquantel Consortium, a global partnership dedicated to developing and delivering a suitable treatment for this vulnerable population, has achieved a significant milestone by administering arpraziquantel, a new oral treatment, to the first preschool-aged child in Uganda. This breakthrough marks a pivotal moment in the global fight against schistosomiasis and offers hope for a healthier future for millions of children.
Arpraziquantel, a 150mg dispersible tablet specifically formulated for children aged three months to six years, addresses the critical need for a palatable and easy-to-administer treatment for young children. The tablet’s dispersible nature allows it to be easily dissolved in water or breast milk, making it suitable for even the youngest patients. Furthermore, its stability in hot and humid climates ensures its efficacy in the regions most affected by schistosomiasis. This innovative formulation overcomes the challenges associated with administering standard praziquantel tablets to young children, which often require crushing and can have an unpleasant taste, leading to poor adherence. The development of arpraziquantel represents a significant advancement in pediatric pharmaceutical formulation and underscores the Consortium’s commitment to ensuring access to appropriate treatment for all affected individuals.
The introduction of arpraziquantel has been met with significant milestones in regulatory approval and international recognition. The European Medicines Agency issued a positive scientific opinion on the drug in December 2023, paving the way for its broader adoption. In May 2024, the World Health Organization (WHO) added arpraziquantel to its List of Prequalified Medicines, a crucial step towards ensuring its quality, safety, and efficacy. The WHO’s prequalification process is a rigorous assessment that evaluates the quality, safety, and efficacy of medicines, particularly those intended for use in low- and middle-income countries. This listing facilitates procurement by international organizations and countries, ensuring wider access to essential medicines. Furthermore, arpraziquantel is expected to be included in the WHO’s List of Essential Medicines Products later this year, further solidifying its status as a critical tool in the global fight against schistosomiasis.
Uganda, a country significantly impacted by schistosomiasis, has taken a leading role in introducing arpraziquantel to preschool-aged children. The first doses were administered as part of the Consortium’s ADOPT (Access and Delivery of Pediatric Treatment) programme, a comprehensive initiative designed to integrate the new treatment into existing healthcare platforms. The ADOPT programme not only focuses on administering the treatment but also on strengthening healthcare systems, training healthcare workers, and raising community awareness about schistosomiasis prevention and control. This integrated approach ensures that the introduction of arpraziquantel is sustainable and contributes to long-term improvements in public health. Uganda’s pioneering efforts will serve as a valuable model for other endemic countries, demonstrating the feasibility and impact of expanding treatment access to young children.
The rollout of arpraziquantel in Uganda is strategically aligned with the WHO’s roadmap for the control and elimination of neglected tropical diseases by 2030. This roadmap outlines a comprehensive strategy for tackling these debilitating diseases, including schistosomiasis, through integrated interventions focusing on prevention, treatment, and morbidity management. The introduction of arpraziquantel for preschool-aged children represents a significant step towards achieving the WHO’s ambitious goals. By addressing the previously unmet treatment needs of this vulnerable population, the Consortium is contributing to global efforts to reduce the burden of schistosomiasis and improve the health and well-being of millions of children. The lessons learned from Uganda’s experience will be invaluable in guiding the expansion of arpraziquantel treatment to other endemic countries.
Following the successful introduction in Uganda, the Pediatric Praziquantel Consortium plans to expand access to arpraziquantel to other schistosomiasis-endemic countries, including Côte d’Ivoire, Kenya, Tanzania, and Senegal. The Consortium is actively collaborating with local health ministries, international organizations, and community partners to ensure equitable and sustainable access to the new treatment. This collaborative approach ensures that the introduction of arpraziquantel is tailored to the specific context of each country and integrates seamlessly into existing healthcare systems. The Consortium’s commitment to providing the treatment on an at-cost basis further underscores its dedication to promoting public health and achieving the United Nations Sustainable Development Goals, particularly SDG 3 (Good Health and Well-being) and SDG 17 (Partnerships for the Goals). The availability of a safe, effective, and affordable treatment for preschool-aged children marks a significant breakthrough in the fight against schistosomiasis and offers hope for a future free from the debilitating effects of this neglected tropical disease.
