P345: Regulatory Considerations in Pediatric Formulation Development for Tropical Diseases on the Example of Emodepside
Poster Presenter
Stefanie Alsing
RA CMC Strategist
Bayer Germany
Objectives
The aim is to explore the regulatory framework to develop pediatric formulations for the anthelmintic emodepside for the treatment of onchocerciasis and soil-transmitted helminth diseases, highlighting challenges and opportunities to ensure child-appropriate medicines in endemic regions.
Method
A review of international and regional regulatory frameworks, including FDA, WHO, and EMA as well as regional guidelines. The analysis is focused on requirements that are relevant to pediatric formulation development specifically addressing the challenges associated with neglected tropical diseases.
Results
Tropical diseases are a major global health burden, particularly in children. Yet suitable pediatric formulations remain limited.
The anthelmintic emodepside is currently under development for the treatment of onchocerciasis and soil transmitted helminths diseases in humans. Given that affected populations are predominantly children, it is crucial to develop safe, effective, and child-appropriate formulations. This, however, requires careful navigation of regulatory frameworks, particularly in the context of neglected tropical diseases, where commercial incentives are limited.
• Regulatory frameworks from authorities including FDA, WHO, EMA and regulatory bodies in endemic countries were considered.
• Challenges include pediatric-specific needs, drug product specific characteristics as well as challenges in the neglected tropical diseases context, including the development of oral solid dosage forms tailored to different age groups, addressing palatability, swallowability, excipient safety, drug product stability in the tropical region, finding a simple suitable formulation that increases the solubility and bioavailability for the low soluble drug substance, limited commercial incentives, reliance on public–private partnerships, and logistical difficulties in conducting pediatric clinical trials in the endemic regions.
• Opportunities exist to streamline development and approval through orphan drug designations, priority review vouchers, WHO Prequalification, reliance pathways, and collaborative procedures (e.g., African Medicines Agency, EUM4ALL, WHO Collaborative Procedure).
Conclusion
We aim to provide a better understanding of the regulatory requirements and their impact on formulation development for pediatric patients. The case of emodepside illustrates the complexity of pediatric drug development for tropical diseases, where scientific and regulatory aspects converge. Early integration of regulatory strategy, alignment across agencies, and tailor-made pediatric formulation development considering regulatory requirements are essential to ensure children affected by helminthic diseases gain equitable access to safe and effective therapies.
