Advanced Therapy Medicinal Products: Recent US Approvals, Upcoming Guidance

By Nicole Gallo and Mamta Puri-Lechner, Ph.D.

Advanced Therapy Medicinal Products (ATMPs) offer promising curative options for treating diseases and injuries. Yet, the development of these therapies involves intricate tactical, procedural, and logistical considerations. While new guidance documents, workshops, and forums aim to enhance ATMP development, the need for harmonization persists to expedite product availability.

This article explores the 2022-2023 approvals of ATMPs by the Center for Biologics Evaluation and Review (CBER) and the newly named Office of Therapeutic Products (OTP). It also delves into the influence of finalized FDA guidances on ATMP development and how town hall meetings can address challenges and optimize opportunities for future ATMP development.

Notably, in September 2022, the FDA restructured the Office of Tissues and Advanced Therapies (OTAT) into the Office of Therapeutic Products (OTP) within CBER. This change aimed to address the evolving landscape of health threats and enhance alignment, review capabilities, and expertise in new cell and gene therapies through structural adjustments and increased staff. The new structure became effective on September 16, 2022, signifying a proactive step in responding to scientific, medical, and regulatory challenges.