The US Food and Drug Administration (FDA) will soon be issuing guidance on its platform technology designation program, which should clear up many industry questions related to the program, asserted Phillip Kurs, regulatory counsel for FDA’s Center for Biologics Evaluation and Research (CBER).
During a session at the CASSS Well-Characterized Biotechnology Products (WCBP) conference in Washington, DC on Monday, Kurs said the new designation program also formalizes the existing pathway for getting products made on novel platforms approved. The goal of the program is to facilitate the development of platform technologies that can be used to manufacture more than one drug or biological product through a standardized production or manufacturing process or processes.
Kurs said the forthcoming guidance should address some of the questions raised at the meeting, including the number of products deemed necessary to designate a platform technology, whether the program is necessary, and the level of documentation that should be submitted to get a platform technology approved. However, he declined to offer a timetable for when the guidance will be issued.
He added that the agency is still on a learning curve with the program, as it is just getting off the ground. “We’re still working on the draft guidance and there will probably be a learning process where sponsors get more familiar with the types of information and the types of leveraging that can lead to efficiencies … I do think that we start out with a 1.0 version of this program that develops over time, and then a version 2.0 and a 3.0 version. I think everyone will have to be patient as we go through this process.”
“It’s a very opportune time for the designation program to be put in into the statue, especially with the lessons of the CRISPR product. The field is rife with opportunities where you may change one insert but otherwise leave the platform virtually unchanged. We are very excited to see how this works,” Kurs said.
In early December, FDA approved Vertex Pharmaceuticals and CRISPR Therapeutics Casgevy, a gene editing treatment for sickle cell disease, and the first approved treatment to use this novel genome editing technology.
Agency asked about need for program
Sarah Kennett, executive portfolio director, pharma technical regulatory at Genentech, asked Kurs about the benefits of applying for platform designation, since manufacturers can already request use of a platform technology from FDA.
Kurs responded that “I think that at a high level, it’s not so much that you were not able to leverage prior knowledge, but it provides a more formal way of doing that. At least in my mind it provides the potential of providing more predictability of the process in the future.”
Mark Schenerman, graduate program director and professor of biotechnology at the University of Maryland Baltimore County (UMBC), asked Kurs to specify the number of products that are needed to be eligible for designation.
Kurs responded that “my guess is that [a forthcoming guidance] would probably say that this is not a magical number and would probably be dependent on what you are trying to do. In general, we try to stay away from using magical numbers.”
The session’s chair, Karen Rule of Pfizer, asked Kurs to address what level of documentation needed support a request for a platform designation, and whether applicants could simply reference some of the technologies used in a prior application or need to provide more in-depth data.
Kurs said that “I think ultimately the onus will be on the applicants to demonstrate that they qualify for the platform designations program,” and that sponsors would need to provide more in-depth data to support the designation.
To continue reading this article please go to RAPS .