Back to: FDA Clinical Investigator Training Course (CITC) 2024
Presenter
Nicole Verdun, MD
Super Office Director
Office of Therapeutics
Center for Drug Evaluation and Research (CDER)
US Food and Drug Administration (FDA)
Dr. Verdun (ver-done) received her undergraduate degree from Duke University and her medical degree from the University of Chicago Pritzker School of Medicine. She then completed a Pediatrics Residency at Children’s Memorial Hospital-Northwestern University and a Pediatric Hematology-Oncology Fellowship at the Children’s Hospital of Philadelphia (CHOP). After practicing as a hematologist with a focus on hemostasis and thrombosis, Dr. Verdun joined FDA in 2012, first in the Office of Hematology Oncology Products as a medical officer and a liaison for sickle cell therapeutics and anticoagulants, and then Therapeutic Biosimilars. She was appointed as the Deputy Director of the Office of Blood Research and Review in the Center for Biologics Evaluation and Research (CBER) in October 2016 and was promoted to Office Director in 2018. In 2023, Dr. Verdun was selected as the Super Office Director of the Office of Therapeutic Products, overseeing 6 Offices dedicated to the regulation and approval of Cell and Gene therapies in the United States. She overseas both a research and regulatory portfolio in CBER.
Abstract
During the 2024 Clinical Investigator Training Course (CITC) virtual conference, Nicole Verdun, Super Office Director in FDA’s Office of Therapeutic Products (OTP) within the Center for Biologics Evaluation and Research (CBER), discusses innovative gene therapies and their regulation. She explains CBER’s mission to ensure the safety and effectiveness of biological products, including gene therapies. The OTP, established in March 2023, aims to enhance expertise and review capacity for cell and gene therapies. OTP regulates a diverse array of gene therapy products, delivered either directly to the subject (in vivo) or by modifying cells outside the body (ex vivo). Verdun highlights key differences in gene therapy development, including new manufacturing technologies, invasive administration procedures, potential long-lasting effects, and risks of unintended DNA changes. Regulatory considerations span complex chemistry, manufacturing, and control (CMC), prolonged biological activity requiring long-term follow-up, and specific preclinical and clinical development aspects. She notes regulatory flexibility for rare diseases, including the use of single-arm studies for efficacy. The FDA utilizes expedited development programs such as Fast Track, Breakthrough Therapy, and the CBER-specific Regenerative Medicine Advanced Therapy (RMAT) designation to address unmet medical needs. Verdun points out the tremendous growth in gene therapy approvals, with 22 products approved to date, mostly for rare disorders. CBER’s 2024 priorities include advancing development for rare diseases, supported by initiatives like the CoGenT global pilot and the START pilot program for therapies addressing life-threatening rare diseases.