The FDA issued draft guidance outlining a new “platform designation” that companies can apply for as part of a broader initiative to modernize the agency’s approach to medicines developed using cutting-edge genetic technologies.

This guidance proves advantageous in areas where multiple therapies can be derived from a single backbone technology. For instance, companies might address different genetic disorders by delivering varying genes via the same engineered virus. A platform designation would streamline the process by allowing a company to leverage data from their first approved gene therapy product—such as manufacturing analyses, inspections, or specific animal safety studies—when seeking approval for subsequent drugs, rather than having to regenerate all data from scratch.

In addition to viral gene therapies, the FDA notes this designation could extend to platforms employing antibodies, siRNA (as used in Alnylam’s drugs), and lipid nanoparticles—the fatty bubbles utilized to deliver mRNA for COVID-19 vaccines and now being explored for gene editing and other rare disease treatments. This new designation represents one of several initiatives proposed by FDA biologics chief Peter Marks to accelerate the development of novel drugs, particularly those targeting very rare diseases.

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