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Session 7 Track 1: Clinical Pharmacology Considerations in the Development and Approval of Oligonucleotide Therapeutics
Session Chair(s)
Hobart Rogers, PharmD, PhD
Pharmacologist
FDA, United States
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)
An Assessment of Immunogenicity and Cardiac Electrophysiology of Oligonucleotide Therapeutics: An FDA Perspective
Hobart Rogers, PharmD, PhD
FDA, United States
Pharmacologist
Oligonucleotide Therapeutics: Assessment of Organ Impairment and Drug-Drug Interactions
Anuradha Ramamoorthy, PharmD, PhD
FDA, United States
Policy Lead, OCP, OTS, CDER
Clinical Development of Lumasiran, the First-Approved Pediatric RNAi Therapeutic
Gabriel J. Robbie, PhD
Alnylam Pharmaceuticals, Inc., United States
Senior Vice President, Clinical Pharmacology and Pharmacometrics
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