Overview: CAR-T FDA approval pathway — context, definitions, and current market posture.
What is CAR-T FDA approval pathway?
The CAR-T FDA approval pathway begins with an Investigational New Drug (IND) application, supported by comprehensive preclinical safety and efficacy data. Once IND clearance is granted, CAR-T therapies advance through phased clinical trials (Phase I–III), during which Good Clinical Practice (GCP) and Chemistry, Manufacturing & Controls (CMC) compliance are rigorously evaluated. The FDA offers expedited pathways—such as Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval—for CAR-T candidates addressing high‐unmet needs in oncology and rare diseases. Successful trial outcomes lead to a Biologics License Application (BLA), after which the FDA conducts a detailed review, inspection of manufacturing facilities, and post-marketing surveillance to ensure ongoing safety and efficacy.
Market snapshot
Current projections indicate the global gene therapy market is expected to reach an estimated $68 billion by 2033, reflecting a base-case Compound Annual Growth Rate (CAGR) of 21.2% from 2025. Within this, the CAR-T therapy market segment alone is forecast to achieve $21.5 billion by 2033. This growth is underpinned by a burgeoning clinical pipeline, with 1,420 active gene therapy clinical trials in 2025, projected to surpass 2,100 by 2033. Oncology represents the dominant therapeutic area, accounting for 64% of global gene therapy trials.
Regulatory notes
Next: See the Biopharma & Life Sciences guide or the full 2025–2033 report for forecasts and detailed methodology.
