CBER Director Offers a Peek Into the Complexities of Where the Center is Headed

Regulatory NewsRegulatory News | 20 June 2017 |  By 

In addition to protecting the US blood supply and ensuring vaccines are safe and effective, the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) is also now regulating complex gene and cell therapies, as well as a new category of treatments: regenerative medicines.

Peter Marks, director of CBER, offered attendees of DIA’s annual conference on Tuesday a few examples of the changing landscape for the center, noting that with the advent of CRISPR/Cas9 genome editing technology, the potential for new therapies is rapidly growing.

Thanks to the 21st Century Cures Act, FDA now has a new designation for regenerative medicines, known as the regenerative medicine advanced therapy (RMAT) designation. As of last week, Marks said there have been 19 requests for RMAT designations, 18 of which CBER has acted on, and four of which have been granted, including the first in May.

Hemophilia A

He also offered the example of hemophilia A treatments, which have progressed from blood transfusions in the 1930s to a possibly curative gene therapy, which he said he hopes could win approval before his retirement.

But one question he brought up momentarily: How much would such a gene therapy, especially if it was a cure, cost?

Right now, Marks said, a year’s worth of hemophilia A treatment can cost between $100,000 and $150,000, so one can imagine how expensive a gene therapy might cost.

“Thankfully at FDA we don’t worry about the price,” Marks said, noting that if a company “can cure it once and for all, it will be an interesting value proposal.”

As media reportshave shown, the prospects for a possibly curative hemophilia gene therapy are rising.


Marks also touched on the rapidly growing field of chimeric antigen receptor-T cells among biotech companies, noting the potential advantages and challenges.



And as far as FDA’s databases of CAR-T information, which have been evaluating the safety of these new therapies for more than a year, Marks said there are currently more than 100 INDs being tracked, many of which target the CD19 antigen. He said the tracking of the safety of such treatments is a good example of FDA being able to evaluate a rapidly developing field.


© 2022 Regulatory Affairs Professionals Society.

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