The reported dismissal of high-ranking CBER officials Nicole Verdun and Rachael Anatol reignited concerns about how gene therapies will be regulated under new FDA leadership.
In the wake of the high-profile departure of Peter Marks, M.D., Ph.D., the FDA has a new leader for its Center for Biologics Evaluation and Research (CBER).
FDA Commissioner Marty Makary, M.D., has named Vinay Prasad, M.D., to the post, he said in a post on X.
Celia Witten, M.D., Ph.D., is no longer deputy director of the FDA’s Center for Biologics Evaluation and Research (CBER), joining the long list of health agency leaders who have departed since President Donald Trump began his second term in office.
In a federal register post Monday, the Office of Therapeutic Products (OTP) of the FDA’s Center for Biologics Evaluation and Research (CBER) unveiled its Cellular and Gene Therapies Interactive Site Tours Program. The initiative is designed to let CBER project managers and reviewers swap regulatory know-how with their industry counterparts on-site.
Accelerated approval—controversial, yes—but absolutely necessary for small, pre-revenue biotechs trying to find new treatments for small patient populations.
That's according to the FDA’s Peter Marks, M.D., Ph.D., who was speaking at the American Society of Gene and Cell Therapy annual meeting Wednesday.
The release of added context on the benefit-risk profile of CAR-T therapies underscores how safety is moving from the periphery into the category’s forefront.
Rare diseases, by their very nature, don’t fit the mold—so neither should the trials for therapies designed to treat them nor the regulatory process to approve them. This was the tone set during a recent panel discussion where Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, argued that non-randomized, single-arm trials could be the best option when testing certain gene therapies for rare diseases.
The director of the FDA’s Center for Biologics Evaluation and Research (CBER) Peter Marks said he’s reasonably comfortable with the progress being made to fill up to 500 vacancies at the agency’s Office of Therapeutic Products (OTP).
Peter Marks, M.D., Ph.D., says he trusts staff reviewers at the FDA, who do “incredibly great work”—but sometimes decisions about efficacy require a broader view.