Nice to read:Gene &Cell Therapy >> Accelerated approval will be...

Nice to read:

Gene &Cell Therapy >> Accelerated approval will be 'the norm' for gene therapies,
FDA's Peter Marks says: The FDA will increasingly use accelerated approval for gene therapies aimed at rare conditions, according to Peter Marks, the FDA’s top official overseeing cell and gene therapies, the clearest signal yet of how widely the agency will deploy its expedited pathway.


Marks’ comments, made last week at a workshop in Washington, build on a stance he took last year backing accelerated approval for gene therapies, which often target young children with ultra-rare conditions.

“If we don’t lean into accelerated approval, we’re going to leave a lot of patients behind,” Marks said during the workshop at the Reagan-Udall Foundation in Washington last week. “Accelerated approval is going to be the norm for a lot of our initial approvals of gene therapies.”

There are more than 10,000 rare diseases, but companies often struggle to bring therapies to market for these conditions due to the small numbers of patients and financial constraints. In recent comments, Marks has indicated that he views accelerated approval as a powerful tool to speed new treatments and ensure that companies don’t give up on promising therapies.

“There are a lot of gene therapies for rare diseases that are almost there,” Marks said. “And it’s a question of, how do we get them across the finish line?”

The agency’s evolving position is part of a wider debate over how to regulate gene therapies, which without regulatory rigor stand to give patients misplaced hope and drain the health system. Confirmatory trials are required as part of the accelerated approval pathway, and typically are randomized controlled trials to verify the clinical benefit. But because these are such small populations with fatal diseases, how these trials are conducted will need to be tweaked given that it would be unethical to run placebo-controlled trials in some of these populations.

The bullish comments from Marks also show how CBER is eager to look at new biomarkers, even if they’re not yet qualified, to condense what would normally be a multiyear approval process. Marks, who announced on Monday that he’s also now the chair of the FDA’s new accelerated approval council to help advance reforms around the accelerated pathway, made clear that the use of biomarkers that have yet to be qualified is already common, and it is not a requirement ahead of an accelerated approval.

“There are plenty of biomarkers that have been used over the years” that were not qualified, he told the workshop. “As long as you can show that you have a biomarker that can be measured accurately and reproducibly, we don’t necessarily always need all of the additional work that gets to qualification,” he said.