DIA/FDA Oligonucleotide-Based Therapeutics Conference

Session Chair(s)

This session will provide timely updates on various important topics pertaining to the clinical pharmacology of oligonucleotides. Specifically, this session will address how FDA regulators evaluate common clinical pharmacology issues relating to assessment of oligonucleotides. In addition, this session will discuss the 2020 FDA approval of OXLUMO (Lumasiran) as the first therapy approved in the US for the treatment of primary hyperoxaluria type 1, a serious, ultra-rare, inherited disease that is usually diagnosed in childhood. This session will review how the key challenges faced during the development were overcome including: establishing a novel surrogate endpoint, pediatric dose selection (encompassing allometric scaling and predictive modeling), and clinical trial design.

Learning Objective :

• Identify common clinical pharmacology challenges in the development of oligonucleotides
• Apply concepts of allometric scaling and population PK/PD modeling to dose selection in unstudied populations
• Translate key learnings from the OXLUMO development program to advance the development of oligonucleotide therapies for other rare diseases and/or pediatric diseases

Speaker(s)