FDA Guidance on Platform Technology Designation Program for Drug Development

Authored by: Christian Cobaugh, Grant Henderson and Khaled Yamout

The FDA’s new guidance clarifies eligible Platform Technologies must adhere to the statutory definition and require consistent CMC processes. Applicants can recycle features across products, while those lacking assets can leverage CDMOs like Vernal Biosciences for standardized processes. Vernal’s CEO, Christian Cobaugh, Senior Director Commercial & Technical Operations, Grant Henderson and guest contributor, Khaled Yamout share their thoughts in this blog article.

Navigating the New Draft FDA Guidance on Platform Technologies 

The FDA’s draft guidance, “Platform Technlology Designation Program for Drug Development,” aims to bring clarity to the process of designating and referencing platform technologies within regulatory filings. 

Defining Platform Technologies 

Acknowledging the inconsistent use of the term Platform, the FDA recently clarified that the Platform Technology designation applies to Drug Products and must comply with the statutory (506K of the FD&C Act) definition, which includes the following: 

“a platform technology is a well understood and reproducible technology, which can 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, where it: (1) is incorporated in or utilized by a drug and is essential to the structure or function of such drug; (2) can be adapted for, incorporated into, or utilized by, more than one drug sharing common structural elements; and (3) facilitates the manufacture or development of more than one drug through a standardized production or manufacturing process or processes.” 

The guidance emphasizes that manufacturing Drug Products under the Platform Technology designation, the manufacturing process must be a standardized process used by an approved drug and useful for structurally similar drugs.  For mRNA medicine manufacturing, examples of Platform Technologies could include shared sequence features such as untranslated regions and poly(A) tail and shared lipids and common lipid ratios that make up lipid nanoparticles.  These shared features of the Drug Products must enable common or standardized manufacturing processes, including raw materials, process parameters, and quality control testing. 

Motivation for Applicants 

The Platform Technology designation has motivated applicants to reuse structural and functional features of products and CMC strategies for multiple assets.  For sponsors that have multiple assets built off a “reproducible technology”, such as a “nucleic acid sequence” or excipients (e.g. lipids), use of the Platform Technology designation is straightforward and facilitated by Change Management under a Pharmaceutical Quality System.  CDMOs, such as Vernal Biosciences, that invest in standardized processes and analytics for drug product classes such as LNP-mRNA, are uniquely positioned to reduce designated Platform Technologies to practice from a manufacturing perspective.  This will require that sponsors have harmonized around the structural and functional features that comprise the definition of Platform Technology.  This approach will allow for faster approvals and lower costs. 

Vernal Biosciences’ Approach 

To democratize access to the Platform Technology designation, Vernal Biosciences has implemented an innovative Change Management system that brings version control to templated, product-agnostic documentation that has established a generalizable CMC strategy while that maintains flexible, execution-level documentation supported by product-specific studies. Under our system, departures from standard procedures are documented, well understood, and easily communicated. 

The Path Forward 

The more sponsors embrace shared access to data and descriptions of standardized processes and methods, the more time and money all parties will save through the use of the Platform Technologies designation, as intended by the FDA. Without this cooperation, the advantages will primarily benefit companies specializing in mRNA with multiple assets as opposed to companies with fewer mRNA assets. 

In summary, while the new FDA guidance on Platform Technologies brings much-needed clarity, it also draws attention to inefficiencies resulting from lack of collaboration and standardization in the industry. By working with the mRNA experts at Vernal Biosciences, the industry can fully realize the benefits of Platform Technologies. 

About the Authors

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