The Global Health Innovative Technology (GHIT) Fund has announced a major milestone with the first administration of arpraziquantel to preschool-aged children in Uganda, marking a significant step in the fight against schistosomiasis. This new pediatric treatment, developed by the GHIT-funded Pediatric Praziquantel Consortium, is being introduced through the ADOPT program, which focuses on integrating the drug into existing healthcare systems in countries where schistosomiasis is common. This achievement also represents the GHIT Fund’s first supported innovation to reach those in need since its inception in 2013.
Schistosomiasis, also known as bilharzia, affects 250 million people globally, with an estimated 50 million preschool-aged children, mainly in sub-Saharan Africa, at risk. Left untreated, the disease can cause severe health issues such as anemia, stunted growth, cognitive impairment, and chronic organ damage, which may even be fatal. Before the development of arpraziquantel, no child-friendly treatment was available for preschool-aged children, leaving many at risk of lifelong health complications.
The Pediatric Praziquantel Consortium developed arpraziquantel to address this gap. This new formulation, designed specifically for young children, offers a climate-stable, water-dispersible, and palatable tablet. It was initially developed by Astellas Pharma Inc. in Japan, with Merck optimizing the formulation in Germany. The clinical trial supply was produced by Merck and Farmanguinhos in Brazil, with current manufacturing done by Farmanguinhos. Future large-scale production is planned to be carried out by Universal Corporation Ltd. in Kenya, ensuring local production to meet the needs of Africa.
The development of arpraziquantel is a direct result of international collaboration, showcasing the power of global partnerships. According to Osamu Kunii, CEO of the GHIT Fund, this achievement highlights the transformative impact of collaboration, bringing together diverse stakeholders to turn what once seemed impossible into reality. The success of arpraziquantel is not just a scientific breakthrough but a reflection of the collective effort to address a critical public health challenge.
Arpraziquantel’s introduction to Uganda is part of a broader implementation program supported by GHIT, which is co-funding the ADOPT program alongside the European & Developing Countries Clinical Trials Partnership (EDCTP). The program aims to assess different implementation models in countries like Côte d’Ivoire, Uganda, and Kenya. It also involves preparing for the introduction of the drug in additional countries, including Senegal and Tanzania.
The successful rollout of arpraziquantel is a vital investment in public health, especially for young children, who are at the highest risk of schistosomiasis. Early intervention is essential to prevent long-term complications like organ damage and cognitive impairment, ultimately reducing the strain on healthcare systems and improving societal well-being. The GHIT Fund and the Pediatric Praziquantel Consortium have worked diligently to ensure that this new treatment can be distributed effectively and equitably to those who need it most.
In December 2023, arpraziquantel received a positive scientific opinion from the European Medicines Agency, and in May 2024, it was included in the World Health Organization’s List of Prequalified Medicines. The next milestone expected is its inclusion in the WHO’s Essential Medicines List in 2025.
Schistosomiasis remains one of the most significant neglected tropical diseases (NTDs), particularly in regions where access to clean water is limited. The disease is transmitted through contact with contaminated freshwater, where the larvae of parasitic flatworms enter the skin and spread to internal organs. In children, the infection can lead to severe long-term health problems, making the development of pediatric treatments like arpraziquantel an urgent priority for global health.
The introduction of arpraziquantel to preschool-aged children represents a major advancement in the fight against schistosomiasis, addressing a critical gap in the treatment landscape and offering hope for millions of vulnerable children worldwide.
