Tatiane Sandes (Farmanguinhos/Fiocruz)
The Institute of Drug Technology (Farmanguinhos/Fiocruz) is closer to starting Pediatric Praziquantel production, indicated for schistosomiasis, a parasitic disease that affects about 200 million people worldwide, half of whom are children. Result of the International Pediatric Praziquantel Consortium, the drug is in the clinical trials last phase. Pilot batches are expected to be produced in March 2022 and the product available in 2024.
It is a water-dispersible tablet, which facilitates its administration to children under the age of six. Until now, the medication available for this disease has not had a version suitable for this audience, which has made it difficult to treat the disease that can lead to anemia, rickets, impaired learning ability, and organs chronic inflammation, which can be fatal in the most severe cases.
Project manager at the unit, Daniel Lacerda reports that the development process for pediatric Praziquantel (PZQ) is in its final phase. "The phase III, clinical studies, which were being conducted in Kenya and Ivory Coast, have finished the recruitment stage and the product administration to the patients. Now it has entered the reconciliation stage and final analysis of all the results so that we can close the report, which is planned for the next year's beginning. A preliminary analysis has shown good results, but we have to wait for the final report for confirmation and full disclosure", he ponders.
In parallel, Farmanguinhos/Fiocruz is in the phase of finalizing the requirements and all the quality documentation necessary to manufacture the product validation batches, essential to compose the dossier to be submitted to the European Regulatory Agency (EMA) and to the National Health Surveillance Agency (Anvisa) to obtain the drug registration, in addition to pre-qualification with the World Health Organization (WHO).
Inspection of facilities
In this sense, the Institute received a visit from the German partner Merck KgaA, the project coordinator, which also involves other international partners, to audit the Technological Complex of Medicines. In the occasion, the facilities and the Quality Management System were evaluated, with a view to the validation batches manufacturing for product registration with Brazilian Health Regulatory Agency (Anvisa, in the Portuguese acronym), expected at the end of this year.
According to the Quality Management deputy director, Rodrigo Fonseca, this is the second inspection within the Consortium. He highlights the Farmanguinhos evolution in this international evaluation dynamic. "This audit type has a high level of demand, especially because the project is international and the partner company, being German, examined us with European requirements. This approach allows our institution to review its procedures and develop itself. Compared to the first visit, in 2018, we saw how much Farmanguinhos has evolved, managing to remedy the points and raising the unit's standards even higher", says Fonseca.
By participating in the International Pediatric Praziquantel Consortium, Farmanguinhos/Fiocruz contributes to this disease eradication that affects millions of people worldwide. Thus, it fulfills not only its institutional mission, but also one of its vision expectations, which is to be recognized by national and international organizations as a strategic center for technological development and drug production.
Recently, the study that evaluated the taste of the existing Praziquantel formulations (Rac-Praziquantel ODT and L-Praziquantel ODT) at the beginning of the project (phase I) was published and served as the basis for the necessary adaptations during drug development. The article was published in the journal PLOS Neglected Tropical Diseases.
Established in July 2012 as the first international not-for-profit public-private partnership related to the fight against schistosomiasis, the Pediatric Praziquantel Consortium is composed of Farmanguinhos/Fiocruz, Merck KGaA (Germany), Astellas Pharma Inc. (Japan), Swiss TPH (Switzerland), Lygature (Netherlands), SCI (United Kingdom), Kenry (Kenya), and Université Félix Houphouët-Boigny (Ivory Coast), and the project is funded by the Bill & Melinda Gates Foundation (United States), Global Health Innovative Technology Fund (Japan), and European & Developing Countries Trials Partnership (Europe). Learn more about the initiative.