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Bethesda North Marriott Hotel and Conference Center

Apr 25, 2022 7:00 AM - Apr 27, 2022 12:00 PM

5701 Marinelli Road, , North Bethesda, MD 20852 , USA

DIA/FDA Oligonucleotide-Based Therapeutics Conference

DIA Your Way! Join us live in-person or live from the comfort of your home/office. This event offers two learning avenues depending on your preference!

Session 7 Track 1: Clinical Pharmacology Considerations in the Development and Approval of Oligonucleotide Therapeutics

Session Chair(s)

Hobart  Rogers, PharmD, PhD

Hobart Rogers, PharmD, PhD


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


Hobart  Rogers, PharmD, PhD

An Assessment of Immunogenicity and Cardiac Electrophysiology of Oligonucleotide Therapeutics: An FDA Perspective

Hobart Rogers, PharmD, PhD

FDA, United States


Anuradha  Ramamoorthy, PharmD, PhD

Oligonucleotide Therapeutics: Assessment of Organ Impairment and Drug-Drug Interactions

Anuradha Ramamoorthy, PharmD, PhD

FDA, United States

Policy Lead, OCP, OTS, CDER

Gabriel J. Robbie, PhD

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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