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First medicine against schistosomiasis for pre-school children

What it is

Arpraziquantel is the first child-friendly medicine in a dispersible formulation to treat and cure schistosomiasis. It is easy to administer and adapted for rural and tropical settings, offering high efficacy with minimal side effects.

Why it matters

Schistosomiasis affects around 240 million people worldwide and severely harms children’s physical and cognitive development. This innovation could protect and improve the lives of 50 million children currently at risk.

Smiling children

The story

Schistosomiasis, a neglected infectious disease (NID) caused by blood flukes, is a debilitating disease that can be fatal and is linked to liver and kidney failure, bladder cancer, extrauterine pregnancy, anaemia, and stunted growth in children. It affects an estimated 240 million people worldwide, with more than 700 million at risk, predominantly children and women in poor, rural communities.

The Pediatric Praziquantel Consortium, co-funded by EDCTP and the Global Health Innovative Technology Fund (GHIT Fund), has developed arpraziquantel, a schistosomiasis medicine for children (3 months–6 years).[1] The tablet dissolves in water, has improved taste, and is designed to withstand tropical climates, making treatment safer, more effective, and easier to administer. The European Medicines Agency issued a positive scientific opinion in December 2023[2], paving the way for WHO approval the year after and introduction in endemic countries.[3]

In parallel, the ADOPT project,[4] co-funded by EDCTP and GHIT Fund, has supported the rollout of arpraziquantel in Kenya, Uganda, and Côte d'Ivoire, focusing on ensuring equitable and sustainable access to this critical medicine. The evidence generated by the project has also enabled other initiatives to implement arpraziquantel in additional endemic settings. This includes its administration in Zimbabwe, where the paediatric formulation has been integrated into national schistosomiasis control programmes. More recently, Tanzania approved the medicine and started its rollout with a landmark pilot in the Lake Zone region, reaching more than 25,000 young children across Itilima, Sengerema, and Kigoma District Councils[4].

Arpraziquantel exemplifies the success of EDCTP programmes in partnering with public and private actors and leveraging co-investments to deliver global health solutions. Supported by EDCTP, the GHIT Fund, the Government of Japan, the Gates Foundation, and the lead developer Merck KGaA (which also provided the medicine for the initial rollout in endemic countries), this innovation demonstrates how such partnerships are critical to transform science into tangible impact.

Global Health EDCTP3 continues to support efforts to tackle schistosomiasis, including among women[5] and improving implementation strategies.[6] Further work is also needed to strengthen procurement and funding mechanisms for NID treatments, ensuring these essential health technologies remain affordable and accessible across sub-Saharan Africa.
 

Sources:

[1] The Pediatric Praziquantel Consortium | Pediatric Praziquantel Consortium & Completion of Phase III trial paediatric schistosomiasis drug arpraziquantel - EDCTP

[2] New treatment for young children with parasitic disease schistosomiasis | European Medicines Agency (EMA)

[3] WHO includes our new pediatric treatment option for preschoolers with schistosomiasis in list of prequalified medicines | Pediatric Praziquantel Consortium

[4] GHIT and EDCTP co-invest additional €7.8 million in access programme for treatment of schistosomiasis in preschool-aged children - EDCTP

[5] WINGS-4-FGS - Global Health EDCTP3 - European Union

[6] 3SI-CONTROL - Global Health EDCTP3 - European Union