FDA Issues Draft Guidance on Platform Technology Designation Program

Rare Daily Staff

The FDA issued draft guidance on its Platform Technology Designation Program, an effort to speed the development of gene therapies that share significant similarities to ones that have already passed muster with the agency.

The agency said a platform technology is eligible for Platform Technology designation if it is incorporated in an approved drug and preliminary evidence demonstrates that the platform technology has the potential to be incorporated in more than one drug, and data submitted by the same party indicates that incorporation of the platform technology is likely to bring significant efficiencies to the drug development or manufacturing process and to the review process.

In the case of gene therapies, the approach has the potential to shorten the preclinical development time when therapies leverage the same vector.

In listing the potential benefits of the designation, the FDA said it will allow for early interactions with the FDA to discuss the use of the platform technology, provide timely advice, leverage data from a previous product, and leverage certain nonclinical safety data from previous products. It will also allow the agency to consider previous findings from inspections for subsequent marketing applications related to the manufacturing of a product using a designated platform technology.

The agency is accepting comments on the draft guidance up to July 29, 2024. Information on submitting comments can be found in the Federal Register.