Marks Eyes Gene Therapy Development Pilot, Sees Increase in Successful RMAT Applications

Posted on June 09, 2022 | By Joanne S. Eglovitch

FDA’s Peter Marks

Peter Marks, director of the U.S. Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER), attributes an increase in the number of advanced regenerative medicine (RMAT) therapy applications approved for cell therapies and genes to sponsors the blow” of the agency’s expectations for nomination requests.

Marks spoke at the California Separation Science Society (CASSS) June 8 meeting on cell and gene therapies. The meeting took place both in person in Arlington, VA and virtually.

Marks also expressed interest in starting a pilot gene therapy program where sponsors would get real-time feedback from FDA reviewers on their development programs. The pilot would test which approaches work best and which don’t in developing these products. This would help avoid setting regulatory policies for approaches that are not practical.

Slight increase in RMAT acceptance

The RMAT program was created by the 21st Cures of the Century Act in 2016 to accelerate the development and approval of products of cell and genetic theories. To qualify for the RMAT designation, products must be intended to treat serious or life-threatening diseases or conditions, and preliminary evidence must indicate the treatment’s potential to address unmet medical needs. The FDA will respond to designation requests within 60 days. Designated products are also eligible for priority review and expedited approval.

As of March 31, 2022, CBER has received more than 180 designation requests, granted 38% of those requests, and denied 56% of them. A small number of applications have been withdrawn or are currently pending.

Marks said that “one of the interesting trends we are seeing is a slight increase in the number of applications we are able to grant compared to the number of nominations submitted. People seem to understand better what we are looking for, I hope this trend continues.

He added that many of these designations relate to cell therapies, while “some” relate to gene therapies. About half of the RMATs relate to rare diseases, and a number of them relate to rare diseases with an orphan product designation.

Marks said the reason for the rejections had “changed over time” when asked to address the reasons for the rejections at the meeting. The most common reason is that the data generated by the trials do not show a clear treatment difference between patients who were treated with the therapy and those who were not.

“The most common reason for refusal is not because the product is not going to treat the serious disease. The problem is that the data generated is often very difficult to see that there is a clear difference between the few patients who have been processed and those that have not.

Brands interested in the gene therapy pilot program

Marks also expressed interest in establishing a gene therapy product development program in which the FDA would work with sponsors developing these therapies to provide “real-time feedback” on their programs. He said this type of feedback and interaction would be similar to Operation Warp Speed, a federal initiative to accelerate the development and production of COVID-19 vaccines, therapies and diagnostics.

During a Q&A at the end of the panel, Marks said the pilot will aim to address agency capacity issues and shed early light on what approaches are working and what isn’t working beforehand. that the FDA does not implement regulatory processes.

“My reason here for trying to do pilot projects is that no, we don’t have the resources right now to offer the kind of individual services that we would like to be able to do.” Yet the pilot would “show Congress that” yes, we did this pilot and in X number of cases we were able to speed up the development of this product by X percent. “With this pilot, he added, the FDA would be able to “excite Congress about what’s possible if you’re able to have these types of interactions.

CASS meeting

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