To improve its reviewers’ understanding of cell and gene therapy manufacturing, the agency has launched a program that will involve a tour of manufacturing facilities and daily workshops for its staffers.
The FDA has unveiled a new program designed to help its staffers and regulatory reviewers to better understand the manufacturing processes behind cell and gene therapies, according to a notice posted in the Federal Register on Monday.
This new initiative, called the Cellular and Gene Therapies Interactive Site Tours Program, will involve project managers and reviewers touring manufacturing facilities of cell and gene therapy developers to observe the production of these biologics, as well as associated packaging, pathology and toxicology assessments and other regulatory affairs.
Throughout the duration of the program, which could last a few days, the FDA’s staff can also join their industry counterparts in daily workshops, which are primarily geared toward improving the understanding of biological product discovery and development. The workshops will also tackle nonclinical and clinical assessments as well as post-marketing activities.
“The overall benefit to regulatory project managers and reviewers will be exposure to project management, team techniques, and processes employed by the biotechnology industry,” the FDA’s announcement read. “By participating in this program, the regulatory project managers and reviewers will gain a better understanding of industry processes and procedures.”
Importantly, the regulator emphasized that these site visits are not meant to be inspections. “Neither this tour nor any part of the program is intended as a mechanism to inspect, assess, judge, or perform a regulatory function,” the agency wrote.
The FDA is currently inviting companies to participate in the program and to reach out to its office for more information.
The Cellular and Gene Therapies Interactive Site Tours Program comes amid a growing number of submissions—and approvals—for cell and gene therapies. In a June 2023 interview with BioSpace, Simon Alfano, associate partner at McKinsey & Company, said that the industry’s cell and gene pipeline had more than 1,000 candidates in clinical development, and over 3,000 in pre-clinical assessments.
“Based on past clinical attrition rates, this level of clinical activity suggests an acceleration of new cell and gene therapy launches toward the second half of this decade,” Alfano continued.
Since then, the FDA has delivered several big decisions in the space, including the December 2023 double approval of Casgevy (exagamglogene autotemcel) and Lyfgenia (lovotibeglogene autotemcel) for the treatment of sickle cell disease. Last month, the FDA also granted full approval to Sarepta’s Duchenne muscular dystrophy gene therapy Elevidys (delandistrogene moxeparvovec-rokl)—despite a failed confirmatory trial.
Still, there is still a long way to go to realize the promise of cell and gene therapy, particularly as regards resolving questions around safety and manufacturing.
