FDA issues draft guidance to speed CGT development

The FDA published draft guidance intended to accelerate the development of cell and gene therapy products, especially those that use genome editing, by allowing the use of existing scientific and regulatory knowledge.

The guidance, released as part of a PDUFA VII commitment, outlines how sponsors can use publicly available information and established platform knowledge, including CMC data, nonclinical study results and clinical information, to streamline regulatory submissions and expedite product development. Rather than generating new data for every program, sponsors may leverage prior experience from similar products, manufacturing platforms, analytical methods, delivery technologies (such as LNPs), genome-editing components (including guide RNAs), and even certain nonclinical and clinical datasets — provided they can scientifically justify that the products and processes are sufficiently similar.

While the draft guidance specifically focuses on genome editing products, the agency says that some of the recommendations may be applicable to other CGT products, such as AAV vectors, nanoparticle-based gene therapy products, and ex vivo-modified cell-based gene therapies that do not incorporate genome editing. 

The draft guidance is open for public comment for 90 days before being finalized.