Pediatric Formulation Design – From Adult to Children

• Agios’ approach for pediatric formulation design
• Pediatric formulation platform selection
• Palatability and excipient selection
• Manufacturing and packaging consideration

Supplying drug to children in the rare genetic disease area is very important since a lot of patients are being diagnosed at young ages. A medicine can really improve their lives and allow them to live more like their peers at the same age. Therefore, it is important to select an age-appropriate formulation for the pediatric population. This presentation will show case the approach of Agios in the selection of a pediatric platform with considerations include manufacturability, patient acceptance, dosage form swallowability and palatability. In addition, the choice of packaging configuration and distribution strategy of the drug is also key to a successful program due to complexity caused by various doses and age groups.

Curtis (Cheuk-Yui) Leung, Ph.D, Associate Director, CMC Formulations, Agios Pharmaceuticals Inc.