CBER To Conduct Interactive CGT Site Tours to Improve Communication, Share Best Practices
The FDA announced its START Pilot Program in October 2023 with a similar goal.
Peter Marks, MD, PhD
Credit: CBER
The FDA has announced a Cellular and Gene Therapies Interactive Site Tours Program (the Interactive Site Tours Program) to improve communication and share best practices between industry and the regulatory agency.1
The program will see regulatory managers from the Center for Biologics Evaluation and Research (CBER) touring biotechnology manufacturing facilities developing cellular and gene therapy products.
“With this program, CBER intends to enhance review efficiency and quality by providing CBER staff with a better understanding of the biotechnology manufacturing industry and its operations,” the Federal Register notice that the program was announced in stated.1
The notice also encouraged any interested industry biotechnology companies to reach out if interested in the program and emphasized that the tours will not be site inspections. The tours would last a few days and CBER members would observe manufacturing and/or packaging, pathology/toxicology laboratories, and regulatory affairs operations to create an open dialogue between industry and CBER. The program would also include daily workshops to better understand biologic product discovery and development activities.
WATCH NOW: Peter Marks, MD, PhD, on Bringing More Gene Therapies to Patients
The new program fits in with CBER’s, and particularly its director Peter Marks, MD, PhD’s, noted goal of helping streamline biologic product evaluations and regulatory paths to help bring more of these novel therapies to underserved patient populations.
“How can we make gene therapy for rare disorders into a reality?[We need]to address some key things that can get in the way of getting gene therapies to patients, one of them is that manufacturing of these products is still quite expensive and complicated... Another is how to move them through clinical trials in a relatively facile manner, making use of what we have at our disposal at the agency, which is things like use of accelerated approval using surrogate endpoints... in order to get an initial approval, which we then confirm with a clinical endpoint... ultimately showing that there's real clinical benefit,” Marks previously told CGTLive®.
The Interactive Site Tours program is one of several new initiatives the FDA has recently announced in pursuit of this goal. Another such program, the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program, was announced in late 2023 with the goal of speeding up the path to approval for several clinical trials evaluating investigational cell therapies and/or gene therapies for rare disease indications and includes several provisions to this end.2
Myrtelle announced in July 2024 that its investigational gene therapy product, rAAV-Olig001-ASPA (previously known as MYR-101), for the potential treatment of Canavan disease, had been chosen for this program. Canavan disease is a rare and fatal childhood genetic brain disease that occurs as a result of mutations in the ASPA gene and has no available disease-modifying therapies. Myrtelle stated that its program met the criteria for the program as it has shown “clinical benefit for rare diseases with unmet medical needs” and the company has shown “ability to accelerate development to market application”. The program will see increased communication between CBER and Myrtelle with reduced wait time between conversations.3
“Opening the lines of communications beyond traditional meeting pathways provides the opportunity to quickly address development issues that would otherwise delay progression to market application. We are encouraged by the opportunity to facilitate the development of a potential treatment for Canavan children who are without treatment options,” Nancy Barone Kribbs, PhD, senior vice president, Global Regulatory Affairs, Myrtelle, said in a statement at that time.3
rAAV-Olig001-ASPA is being evaluated in a first-in-human, phase 1/2 trial (NCT04833907) that seeks to enroll 24 participants with Canavan disease to receive a single, 3.7 × 1013 vg dose of gene therapy intracerebroventricularly.
