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Development & registration program

The Pediatric Praziquantel Consortium aims to reduce the global disease burden of schistosomiasis by addressing the medical needs of infected preschool-age children. Our objective is to develop, register and provide access to a suitable pediatric medication for treating schistosomiasis in this age group.

The Consortium’s clinical development program was completed with positive results from the pivotal clinical Phase III trial in November 2021. At the end of 2022, the European Medicines Agency (EMA) validated the regulatory application and officially began the review process. In December 2023, the Consortium received a positive scientific opinion from EMA

More information on the EMA positive opinion is available on the EMA website

The pediatric drug development program was divided into three major steps: preclinical development, clinical development and registration:

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    Preclinical development

    Our preclinical program (completed in 2014) focused on the development of a novel child-friendly treatment option based on praziquantel. For more information on the technology behind our potential new pediatric treatment option, please visit our pediatric treatment section

    The preclinical development program consisted of six major activities:

    • Manufacture of the L-praziquantel active pharmaceutical ingredient (API)
    • Development of novel orally dispersible tablet (ODT) formulation candidates
    • Development and validation of analytical and bioanalytical methods
    • Metabolism and pharmacokinetics
    • Toxicology, including safety pharmacology
    • Good Manufacturing Practice (GMP): manufacture and documentation of the new ODT for use in clinical trials.
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    Clinical development

    The clinical development program was set up in line with EMA recommendations for pediatric development. It was designed with the support of regulatory authorities and a panel of international experts, including clinicians from endemic countries. Praziquantel (PZQ) is an existing drug, but the potential new pediatric treatment option and the target age group are new and therefore required a full phase I-III clinical development program:

    • Two phase I bioavailability studies in healthy adult volunteers were performed in South Africa with the aim of determining the pharmacokinetic properties of the Racemate (Rac)-PZQ and L-PZQ formulation candidates in comparison with the current Cesol® 600 mg PZQ commercial racemate tablets. The pharmacokinetics of the two development candidates was characterized and both showed a good safety profile and acceptable palatability. The studies were completed in 2015.
    • swill-and-spit taste study was performed in 2015 in Tanzanian children (age 6-11 years) to compare the “overall palatability” of the two development candidates L-PZQ ODT and Racemate PZQ ODT with the current Cesol® 600 mg PZQ commercial racemate tablets. This study was completed in 2015.
    • phase II dose-finding study was performed in Côte d’Ivoire in S. mansoni-infected children of different ages (range 3 months to 6 years) from 2016-2018. Both development candidates were investigated in this study with the goal to select the right candidate and dose for the phase 3 trial. The study confirmed that L-PZQ ODT would be investigated further in late-stage development due to an improved overall profile compared to Rac-PZQ ODT. It also identified the dose to be investigated in the phase 3 trial.
    • Phase III trial was conducted in Côte d’Ivoire and Kenya between 2019-2021. This study was an open-label, efficacy and safety trial of L-PZQ ODT in Schistosoma-infected children 3 months to 6 years of age, including a 2:1 randomized, controlled cohort of Schistosoma mansoni-infected children 4 to 6 years of age treated with L-PZQ ODT or commercial praziquantel. The primary efficacy endpoint of clinical cure was met with a favorable safety profile.

    All of our clinical trials were conducted according to Good Clinical Practice and applicable ethical guidance and regulations, in order to protect the well-being and rights of adults and children enrolled in the trials. The trials were designed to reflect the public health needs and priorities of the countries in which they were carried out.

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    Registration

    In November 2022, the Consortium submitted its regulatory application to the European Medicines Agency (EMA) under the EU-M4all procedure for high-priority medicines for markets outside the European Union. EMA validated the application and started the review process in December 2022. In December 2023, the Consortium successfully obtained a positive scientific opinion of its new pediatric treatment option from EMA. This decision will be followed by WHO prequalification and local approval in African countries through a collaborative procedure pathway. Apart from sub-Saharan Africa, members of the Consortium aim to submit the Marketing Authorization Application in Brazil.

    For more information on where we stand, please visit our timeline

    More information on the EMA positive opinion is available on the EMA website

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