Five regulatory myths about cell and gene therapy (CGT) orphan drug development

By Steve Winitsky, M.D., Senior Vice President, Technical, Regulatory Consulting

Published on: Jun 1, 2026

6 min

Five regulatory myths about cell and gene therapy (CGT) orphan drug development

In my earlier work at the FDA, and now with Parexel clients, I have encountered several persistent misconceptions about how the agency’s Center for Biologics Evaluation and Research (CBER) and Office of Tissues and Advanced Therapies (recently renamed the Office of Therapeutic Products) view orphan drug-designated cell and gene therapies (CGTs).

Here are five of them: