Gene Therapy Platforms May Require Less Preclinical Data, Gottlieb Says

Thursday, September 7, 2017

US FDA Commissioner Scott Gottlieb said the agency is developing new policies for preclinical development of products involving new technology platforms, whereby certain second entry therapies could require less preclinical data.

"We're going to be taking other new steps to make sure that our policies governing early, pre-clinical science are more closely matched to the complexion of modern technologies," Gottlieb said in a Sept. 7 speech to Research America's National Health Research Forum in Washington, DC.

"One is evidenced in how clinical trials are initially designed. In certain cases, there are a lot of common features across the same platform, even as it's used to target different genes or proteins. There may be a plausible reason to recognize how a product or platform can work across multiple disease states – and leverage the learning from one setting in other opportunities."

One example of FDA acting on these principles came over the summer when Merck & Co. Inc.'s Keytruda (pembrolizumab) became the first cancer treatment to be approved based on a common biomarker rather than the location where the tumor originated.

In another example, "we may be able to take a more adaptive approach in our pre-clinical evaluation of different therapies that share a lot of common characteristics in the overall platform used to deliver a gene product," Gottlieb said. "Consider two gene therapy vectors that contain CRISPR constructs differing by only one base pair. These two products might not need nearly the same amount of pre-clinical data for the second variant as was required for the first one, if we can borrow what we learn across different clinical applications of the same basic
construct." (subscription only)

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