Biologics

CBER in a ‘Transition Year’: Marks

CBER director Peter Marks says 2023 will be a transition year for the Center as it staffs up to do more work on cell and gene therapy and reorganizes its organizational structure and business practices to meet the new demands. Marks discussed the changes during an appearance on Cell & Gene The Podcast.

He encouraged industry members to communicate with CBER early and often and also asked for patience as the Center brings on about 100 people to work directly in cell and gene therapy and another 25 support staff. He also encouraged industry representatives to look for CBER workshops and other outreach efforts in the second half of the year.

Asked what it means to communicate “early” with CBER, especially relating to manufacturing, Marks replied that it would likely be before a company has produced doses to treat humans or has produced doses to treat just a few people. He noted that when the agency gives CMC (chemistry, manufacturing, and controls) advice to companies, it does so from a broader perspective than the company often has, based on what FDA is seeing in other companies. “You certainly can challenge us,” he said, “but understand what FDA’s perspective is and how we know what we know, even though we can’t talk about what we’ve seen in other companies.”

Marks said the greatest change in his time at CBER has been the approval of generically modified cancer therapies. “We are sitting on a large growth curve,” he said.

With CBER staffing up to provide earlier feedback to sponsors on their development programs, Marks encouraged sponsors to plan for success and know early on how they will manufacture and characterize their products.

Asked what CBER learned from the Covid-19 pandemic, Marks spoke about the importance of communication in expediting development. He said the Center will be exploring ways of having more informal meetings with sponsors to encourage greater communication.

The clinical development innovations he expects to see in the next three years are (1) off-the-shelf CAR-T cells that don’t need to be harvested from a sick patient and (2) the potential for directly administered genome editors.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome