Mariangela Longinio (Farmanguinhos/Fiocruz)
The European Medicines Agency (EMA) has adopted a positive scientific opinion for Arpraziquantel for the treatment of schistosomiasis in preschool children (3 months to 6 years old). The application was submitted by the Pediatric Praziquantel Consortium, under the EU-M4all procedure for high-priority medicines for human use intended for countries outside the European Union. In Brazil, the regulatory submission to the Brazilian Health Regulatory Agency (Anvisa) will be carried out by the Consortium's partner, the Institute of Drug Technology (Farmanguinhos/Fiocruz).
The positive scientific opinion issued by the EMA is the basis for the possible inclusion of Arpraziquantel on the World Health Organization's (WHO) list of essential and pre-qualified medicines. Farmanguinhos brings its experience in production and distribution and will be the manufacturing site, after Anvisa's approval, for the future introduction of the new pediatric medicine in endemic countries. By developing, registering and providing access to Arpraziquantel, the Consortium is making a tangible contribution to the elimination of schistosomiasis as a public health problem, in addition to addressing the SDGs, particularly SDG 3 (Good Health and Well-being) and SDG 17 (Partnerships for the Goals).
The current standard of treatment for schistosomiasis is Praziquantel, which is safe, effective and adequate for school-age children and adults. Expanding the range of options to treat schistosomiasis, Arpraziquantel is adapted for preschool children against Schistosoma mansoni and Schistosoma haematobium. Tested in clinical development, Arpraziquantel contains the pharmacologically active enantiomer of Praziquantel. It is a 150 mg dispersible tablet. The prototype of its pediatric formulation was developed by Astellas, in Japan, and later perfected by Merck, in Germany. The manufacturing process was used to produce inputs for clinical trials by Merck and Farmanguinhos/Fiocruz in Brazil.
Future production is to be carried out by Universal Corporation in Kenya, which is preparing for an extensive local production capacity for Africa, and by Farmanguinhos/Fiocruz. When developing Arpraziquantel, the Pediatric Praziquantel Consortium established a development program for pediatric medicine, divided into four main stages: pre-clinical development, clinical development, registration and access. All details can be found on the Consortium's website.
Schistosomiasis (also known as bilharzia) is one of the most prevalent parasitic diseases worldwide with great relevance in terms of its economic impact and public health burden. It is a related disease that is widespread in tropical and subtropical regions where large sectors of the population do not have access to drinkable water. Platyhelminths transmit the disease and people become infected with the parasite through contact with fresh water while working, swimming, fishing or washing clothes, for example. The tiny larvae penetrate the human skin, enter the blood vessels and attack the internal organs. The infection rate is very high among children. Schistosomiasis is a chronic condition, classified by the WHO as a neglected tropical disease.
The EMA, through the EU-M4all procedure, in cooperation with the WHO, can provide scientific opinions on high-priority human medicines, including vaccines, intended for markets outside the European Union (EU). The procedure was previously known as the Article 58 procedure, as its legal basis is Article 58 of Regulation (EC) No. 726/2004.
The Pediatric Praziquantel Consortium is an international public-private partnership that aims to reduce the global stigma of schistosomiasis and improve child health by addressing the medical needs of infected preschool children. Its mission is to develop, register and provide access to a pediatric medicine suitable for the treatment of schistosomiasis in children aged 3 months to 6 years. Funded by Merck, it has in-kind contributions from Consortium partners and grants from the Bill & Melinda Gates Foundation, the Global Health Innovative Technology Fund and the European & Developing Countries Clinical Trials Partnership.
The Consortium's partners are Merck (Germany), Astellas Pharma (Japan), The Swiss Tropical and Public Health Institute (Switzerland), Lygature (Netherlands), Farmanguinhos/Fiocruz, Unlimit Health (United Kingdom), Kenya Medical Research Institute (Kenya), Université Félix Houphouët-Boignen (Côte d'Ivoire), Klinikum rechts der Isar der Technischen Universität München (Germany), Ministry of Health of Côte d'Ivoire (Côte d'Ivoire) and African Institute for Health and Development (Kenya).
Other collaborators contributing to the Pediatric Praziquantel Consortium's mission are the Makerere University School of Public Health (Uganda), the Kenyan Ministry of Health, the Division of Vector-Borne and Neglected Tropical Diseases (Kenya) and the Ugandan Ministry of Health.