Sponsors often struggle with early PIP submissions for pediatric gene therapies due to their complexity. This article highlights EMA’s guidance, the stepwise PIP pilot, and approaches to harmonize global pediatric plans for regulatory success.

The FDA’s Office of Therapeutic Products (OTP) is taking strategic steps to support the gene therapy industry, aiming to approve 10-20 cell and gene therapy (CGT) products annually by 2025. Initiatives like CoGenT and START enhance global collaboration and accelerate rare disease therapeutics development.