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The FDA is spelling out the details of a new pathway to help speed personalized cell and gene therapies to market for rare diseases.
Monday’s long-awaited draft guidance outlines the agency’s “plausible mechanism” framework, a pathway FDA Commissioner Marty Makary and vaccines chief Vinay Prasad unveiled last fall for drug developers pursuing bespoke therapies.
The guidance details a path to market for therapies where a randomized trial is not feasible and where there’s a specific genetic, cellular or molecular abnormality that can be altered or corrected. Genome editing and RNA-based therapies, including antisense oligonucleotides, are the focus of the 22-page draft. But the agency notes that the general concepts may apply to other types of individualized therapies.
“Specifically, this guidance applies when clinical evidence from a limited number of patients will be available to support the individualized product’s safety or efficacy in the intended patient population,” the draft says.
A senior FDA official told reporters on a call on Monday that the guidance will not change the agency’s approval standards. But the guidance notes some significant changes.
For instance, when...
