The FDA on Tuesday released a long-awaited draft guidance explaining how companies can win designations for platform technologies, a regulatory step that could speed up cell or gene therapy approvals based around common technologies.
The 15-page document explains how the designations could help biopharma companies use prior data from the platforms, such as batch and stability data, manufacturing inspection results or nonclinical safety data “such that a product-specific assessment for specific, designated endpoints might not be warranted.”
The designations could be particularly beneficial to gene therapies that might use the same AAV backbone and come from the same company, said Nicole Verdun, head of the FDA’s newly created Office of Therapeutic Products, at a conference last September. The agency only expects about 10 designation requests per year, according to the Federal Register notice.
“There are several potential benefits for a drug company that receives a platform technology designation, including having early and additional interactions with FDA to discuss the use of a platform technology and leveraging information previously submitted in support of the designation in subsequent submissions from the same sponsor, such as new drug or biologics license applications,” CBER spokesperson Lorrie McNeill told Endpoints News in a statement.
FDA defines a platform technology as “a well understood and reproducible technology, which may include a nucleic acid sequence, molecular structure, mechanism of action, delivery method, vector, or a combination of any such technologies that FDA determines to be appropriate.” The agency outlined three specific criteria for what a platform should be, such as “essential to the structure or function of such drug,” and that it “can be adapted for, incorporated into, or utilized by, more than one drug sharing common structural elements,” and that it helps manufacture or develop more than one drug through a standardized process.
FDA pointed to lipid nanoparticle platforms for mRNA vaccines or gene therapy products, lipid nanoparticle platforms for different short, single-stranded or double-stranded oligonucleotides, monoclonal antibody platforms, and more as examples.
A platform technology designation doesn’t mean that companies would lose out on accelerated approvals or other expedited pathways, the agency said.