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 Kenya Medical Research Institute (KEMRI) announced.
According to a press release, the Pediatric Praziquantel Consortium announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive scientific opinion for arpraziquantel to treat the neglected tropical disease, schistosomiasis.
Kenya Medical Research Institute (KEMRI) is a partner member of a consortium involved in the just concluded human research for the treatment of schistosomiasis in preschool-aged children that has shown promising results.
‘‘This positive opinion is not only good news for the pull of scientists involved in the study, but also for targeted preschool-aged children with schistosomiasis globally,’’ reads the statement in part.
Schistosomiasis (commonly known as bilharzia or snail fever) is a neglected tropical disease that affects over 240 million people worldwide. It is one of the most important tropical diseases in terms of public health burden and economic impact.
The existing ‘standard of care’ treatment for schistosomiasis is praziquantel, which in Kenya, is made available through mass drug administration programs to adults and school-aged children. However, the drug is not suitable for children six years of age and younger. As such, around 50 million preschool-aged children currently lack adequate treatment.
The development and introduction of this new pediatric medicine, Arpraziquantel is indeed an unprecedented opportunity to close the treatment gap and break the transmission of schistosomiasis in Kenya.
The application was submitted by Merck, on behalf of the Consortium, under the EU-M4all procedure for high-priority medicines for human use intended for countries outside the European Union.
“After more than 10 years of intense collaboration, we are thrilled to have received a positive scientific opinion from EMA. I am extremely proud of our Consortium of dedicated partners. Together, we have come a long way in our vision of providing a treatment option for the most vulnerable population – the youngest. This will contribute to reducing the global disease burden of schistosomiasis, a neglected tropical disease that affects approximately 240 million people worldwide, now, we all need to turn our full attention to access and delivery,” said Dr. Jutta ReinhardRupp, Chair of the Pediatric Praziquantel Consortium Board and Head of the Global Health Institute at Merck.
KEMRI participated in the Phase III clinical trial of Pediatric Praziquantel (PZQ) conducted in Homabay, Kenya at the Homabay Teaching and Referral Hospital (HTRH). The open-label study evaluated the efficacy and safety of Levo-Praziquantel (l-PZQ) 150mg in Schistosoma infected children 3 months to 6 years of age, and included a 2:1 randomized, controlled cohort of Schistosoma mansoni-infected children 4 to 6 years of age treated with L-PZQ Orodispersible tablets (ODT) or commercially available 600mg praziquantel.
The new drug is a small, orally dissolvable tablet that can withstand the heat and humidity of tropical climates, and has an acceptable taste that will make it tolerable to young children and enhance its pharmacological effectiveness in treating schistosomiasis.
Arpraziquantel is derived from praziquantel, the standard of care treatment for schistosomiasis developed in the 1970s. The tablet is to be administered dissolved in water and along with an improved taste that makes it palatable for very young children. Schistosomiasis transmission in Kenya is still a major public health concern with the Schistosoma parasite endemic in 62 of the 290 sub-counties in Kenya.
There are three major endemic areas – the Coastal region (mainly Schistosoma haematobium parasite that causes the urogenital form of the disease), parts of Central and Lower Eastern areas (both S. haematobium & S. mansoni) and the Lake Victoria basin (mainly S. mansoni parasite that causes the intestinal form of the disease) with few pockets of S. haematobium further inland. Transmission is still commonplace in many areas, with people infected during a wide range of activities such as domestic, agricultural and commercial.
Exposure to the worm can happen while car washing, sand harvesting, and fishing, and during recreational activities such as swimming.
“KEMRI is very committed together with other partners to confirming the potential of the new pediatric PZQ formulation. Innovation is a key driver in finding today’s health solutions, as an Institute we are proud to be part of improved treatments for the close to 250,000 young children in the country that are infected by, one of the Neglected Tropical Diseases (NTDs),” said Prof. Elijah Songok, KEMRI Ag. Director General & CEO.
The positive CHMP scientific opinion by EMA is the basis for the potential inclusion of arpraziquantel into the World Health Organization’s list of prequalified and essential medicinal products. Together with the positive scientific opinion, the planned prequalification will support the regulatory pathway in African 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 and thereby also addressing the SDGs, in particular SDGs 3 (Good Health and Wellbeing) and 17 (Partnerships for the Goals).