Where we stand

First preschool-aged children treated in Uganda. The rollout in Uganda is part of the Consortium’s ADOPT program, aimed at integrating the new pediatric treatment into healthcare systems and preparing for its broader introduction across sub-Saharan Africa, starting with Côte d’Ivoire, Kenya, Tanzania, and Senegal.

The Consortium received WHO prequalification in May 2024.

The Consortium received a positive scientific opinion from EMA in December 2023.

The Consortium submitted the regulatory application to EMA in November 2022 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.

On 18 Sept 2019, the Pediatric Praziquantel Consortium announced that the first patient in a Phase III pediatric clinical trial being conducted in Kenya and Ivory Coast has been dosed.

A supplementary US$ 4.1 million investment from the GHIT Fund has further reinforced the Pediatric Praziquantel Consortium’s efforts to develop, register and provide access to a novel orally dispersible praziquantel tablet formulation for children. It will help the Consortium in its efforts of bringing closer the treatment of preschool-age children suffering from schistosomiasis.

A phase II dose-finding study is being performed in Côte d’Ivoire in S. mansoni-infected children of different ages (range 3 months to 6 years) from 2016-2018. The study part involving the age group 2-6 years was completed in 2017 and confirmed the formulation (L-PZQ) and the dose that will be pursued by the Consortium until registration.

On 8 December, the European & Developing Countries Clinical Trials Partnership (EDCTP) and The Global Health Innovative Technology Fund (GHIT) announced that they will be co-funding the Phase III clinical study conducted by the Pediatric Praziquantel Consortium. 

On 30 March 2017, the GHIT Fund announced that it will continue to support the Pediatric Praziquantel Consortium. 

By sharing experiences from the Consortium, the PLOS NTD Symposium Article highlights the crucial role of expert meetings in PPPs targeting NTDs.

On 16 June 2016, the Consortium announced that he first patient in a Phase II pediatric clinical trial being conducted in the Ivory Coast has been dosed.

The phase I bioavailability studies in healthy adult volunteers were performed in South Africa with the aim to determine the pharmacokinetic properties of the Racemate (Rac)-PZQ and L-PZQ formulation candidates in comparison to the current 600 mg PZQ commercial racemate tablet formulation (Cesol). Both formulation candidates showed a good safety profile and acceptable palatability.

A swill-and-spit taste study was performed in 2015 in Tanzanian school-age children. The study demonstrated that the tastes of the new L-PZQ ODT and the Rac-PZQ ODT tablets were improved compared to the taste of the Cesol 600 mg PZQ commercial racemate tablet formulation.

The Pediatric Praziquantel Consortium, a Consortium dedicated to the development of a new pediatric formulation against schistosomiasis received almost USD4.9 million from the Japanese Global Health Innovative Technology (GHIT) Fund.

A meta-analysis of the use of praziquantel in children has been conducted to guide the clinical development strategy, and has been published in PLOS Neglected Tropical Diseases in 2014.

Tech Transfer of the pediatric formulation processes from Astellas to Farmanguinhos for the racemate PZQ tablets to produce GMP batches for the clinical supplies have been completed.

Regulators from many different African countries took part in a recent Pediatric Praziquantel Consortium meeting, held in Geneva in October. An intensive two-day agenda encompassed discussions of the entire regulatory approach for the Consortium’s new praziquantel pediatric formulation.

The Pediatric Praziquantel Consortium, launched in 2012 as an international non-profit public-private partnership, has announced that it has been awarded a prestigious research grant from the Japanese Global Health Innovative Technology (GHIT) Fund.

Tech Transfers of the pediatric formulation processes from Astellas to Merck for the L-PZQ tablets to produce GMP batches for the clinical supplies have been completed.

A pre-clinical package comparing the racemate PZQ and the L-PZQ was initiated in October 2012 and was completed in November 2013. No toxicity issues have been reported.

The Bill & Melinda Gates Foundation grants $1.15 million to support some research & development activities before Phase I clinical trials. The current partners in this consortium, coordinated by TI Pharma, are Merck, Astellas Pharma Inc. and the Swiss Tropical and Public Health Institute ("Swiss TPH").

In August 2013, a second Expert Meeting was organized, evaluating design of the clinical studies needed to register the new PZQ formulation with the help of international experts, including clinicians from endemic countries.

Soon after its formation, the Consortium collaborated with a panel of experts in schistosomiasis and pediatric formulations, at a meeting in Geneva in August 2012. The goal was to develop a target product profile (TPP) delineating both required and desirable formulation features. Factors taken into account included therapeutic indications, pediatric age group, dosage form, frequency of administration, formulation storage conditions and more.

A newly established international public-private partnership (PPP) between TI Pharma (now Lygature), Merck (Darmstadt, Germany), Astellas Pharma Inc. and the Swiss Tropical and Public Health Institute ("Swiss TPH") has been launched to develop a new pediatric formulation of praziquantel to combat schistosomiasis - commonly known as bilharzia - in preschool children.