The purpose of this research is to analyze regulatory approvals of Cell and Gene Therapy Products by the FDA from 2020 to 2024. The study aims to explore the utilization of expedited regulatory pathways, including RMAT designation, and their impact on the approval trends of cell and gene therapies. Additionally, the research investigates therapeutic area-specific trends and highlights the significance of advanced Therapies in addressing unmet medical needs.

Data on FDA-approved Cell and Gene Therapies (2020–2024) were sourced from the FDA's public database. Approvals were analyzed by regulatory designations and therapeutic areas, with a focus on RMAT designation, introduced in 2016 to expedite advanced therapy approvals.

Between 2020 and 2024, the FDA approved 25 Cell and Gene Therapy products, with 36% approved in 2024. The majority (34%) received Orphan Drug designation, followed by Breakthrough Therapy (20%) and RMAT (16%). Oncology led with 31% of approvals. RMAT requests and approvals increased, reaching a 70.7% approval rate in 2024, reflecting improved submission quality and regulatory alignment. These trends highlight the FDA's focus on innovation while maintaining safety and efficacy standards.

Between 2020 and 2024, 25 ATMPs were approved in the US, showcasing advancements in regenerative medicine, particularly in oncology and rare diseases. Expedited pathways like RMAT, Breakthrough Therapy, and Orphan Drug designations accelerated access while ensuring safety. These trends underscore the role of adaptive regulatory frameworks in driving innovation and addressing critical unmet needs, paving the way for future breakthroughs.