Schistosomiasis is a neglected tropical disease caused by blood flukes (trematode worms) that can remain in the body for many years and cause damage to organs such as the bladder, the kidneys and the liver. The parasite lives in fresh water in tropical and subtropical regions. The disease mostly affects communities without access to safe drinking water and adequate sanitation. It is found throughout Africa, and also in parts of South America, the Caribbean, the Middle East and Asia.
According to the World Health Organization (WHO), more than 250 million people required preventative treatment for schistosomiasis in 2021. At least 20,000 people die from the infection annually. It has been widely noted that the lack of a paediatric formulation suitable for young children, which amount to approximately 50 million patients, has limited the ability to control the disease. In children, schistosomiasis can have severe consequences, including impairment of physical and cognitive development.
Arpraziquantel is the first medicine formulated to meet the needs of preschool-aged children. It is derived from praziquantel, the standard treatment for schistosomiasis developed in the 1970s. Arpraziquantel induces muscular paralysis in the schistosome worms, which causes them to loosen their grip on the wall of mesenteric veins. Instead, they migrate to the liver where they are eventually destroyed and eliminated.
The medicine will be available as 150 mg dispersible tablet. It is approved for children weighing at least 5 kg and it is to be taken fully dispersed in water using a cup or a syringe. The medicine has a taste acceptable for young children and withstands the hot environment of tropical climate.
The benefits and safety of the medicine have been evaluated based on the outcomes from phase II and phase III clinical trials in which 442 children aged 3 months to 6 years received Arpraziquantel. During the trials, very high cure rates were reported as no parasite eggs were observed in the stool or urine weeks after treatment.
The most frequently reported suspected adverse events were abdominal pain, diarrhoea and vomiting.
The development of Arpraziquantel was driven by the Pediatric Praziquantel Consortium, a public-private partnership dedicated to the development of this medicine as a paediatric treatment for schistosomiasis in preschool-aged children. The project is funded through the European and Developing Countries Clinical Trials Partnership (EDCTP) between European and African countries that supports collaborative clinical trials and clinical studies on poverty-related infectious diseases in Africa and the Japan-based Global Health Innovative Technology (GHIT) Fund.
Merck Europe B.V. submitted the application under the EU-M4all procedure for high-priority medicines for human use intended for markets outside the European Union (EU) on behalf of the Pediatric Praziquantel Consortium. EMA’s positive scientific opinion will streamline the WHO prequalification and facilitate national registration of Arpraziquantel for use in public health programmes in countries where the disease is endemic.
Medicines submitted under the EU-M4all programme are assessed by the CHMP in collaboration with the WHO and the target countries, combining EMA’s scientific review capabilities with the epidemiology and local disease expertise of WHO and experts and national regulators in the target countries. The CHMP scientific opinion under the EU-M4All procedure supports global regulatory capacity building and contributes to the protection and promotion of public health beyond the EU by assessing medicines for countries where regulatory capacity may be limited. National regulators can rely on the CHMP's scientific assessment to decide on the use of the medicine in their countries.
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