Designating a Platform Technology: FDA’s Long-Awaited Draft Guidance

In newly released Draft Guidance from the U.S. Food and Drug Administration (FDA) entitled, Platform Technology Designation Program for Drug Development, the FDA addresses its new designation program for platform technologies, which is intended to bring efficiencies to drug development, manufacturing, and review processes for applications that incorporate designated platform technologies.

FDA notes that the definition of a “platform” in this context may differ from how the term is used in the industry, so it clarifies that under the statute, a “platform” is defined as a well-understood and reproducible technology where it: (1) is incorporated in or utilized by a drug or biologic and is essential to the structure or function of such drug or biologic; (2) can be adapted for, incorporated into, or utilized by, more than one drug or biologic sharing common structural elements; and (3) facilitates the manufacture or development of more than one drug or biologic through a standardized production or manufacturing process or processes. Criteria for designation as outlined by statute are as follows: (1) it is incorporated in, or used by, an approved drug or biologic; (2) preliminary evidence demonstrates that the platform technology has the potential to be incorporated in, or used by, more than one drug or biologic without an adverse effect on quality, manufacturing, or safety; and (3) data or information indicates that incorporation or use of the platform technology has a reasonable likelihood to bring significant efficiencies to the product development or manufacturing process and to the review process. FDA identifies the following examples of potential platform technologies: lipid nanoparticle (LNP) platforms for mRNA vaccine or gene therapy products; monoclonal antibody platform technologies; platforms using a chemically defined targeting moiety in conjugation with a well-characterized synthetic siRNA; and LNP platforms encapsulating different short, single stranded or double stranded oligonucleotides.

Importantly, the FDA outlines the following for industry:

• A request for designation of a platform technology should come during the investigational new drug (IND) stage of development for a planned subsequent new drug application (NDA), 351(a) biologics license application (BLA), or request for Emergency Use Authorization (EUA)
• The designation request should include a detailed justification, including summary evidence from completed studies, demonstrating that incorporation or use of the platform technology is reasonably likely to bring significant efficiencies to the review process
• Preliminary evidence includes data and findings from completed tests or studies that evaluate: the proposed use of the platform technology in an already approved drug product; the proposed use of the same platform technology in a new drug product; or the use of a platform technology across scenarios (drawing comparisons between scenarios)
• There should be “minimal differences” between the approved product using the platform technology and the product under investigation that proposes to use the same platform technology
• A designation request should include the following content: identification of a shared structural element between drug products; justification and scientific support for use of a platform technology across products noting how subsequent uses would not affect safety, quality or manufacturing; and a risk assessment evaluating how later uses of the platform technology could affect relevance of the prior information

FDA clarifies that:

• Designation would not confer third parties additional rights of reference to platform technology information unless they own or have full rights to reference that information; an agreement showing a full right of reference should be submitted in Module 1 by any third party seeking to leverage information provided in a prior application related to the designated platform technology
• A BLA holder is expected to have knowledge and control over the manufacturing process for its product; NDA sponsors leveraging platform technology information from prior applications can do so by cross-reference but BLA sponsors must include full information in subsequent applications
• It may take actions to expedite the development and review of applications incorporating designated platform technologies, but this does not mean an expedited action date clock under FDA’s established user fee timelines nor does it have any impact on eligibility for any expedited approval pathways
• It will issue a determination within 90 days of receipt of designation requests; however, a finding of ineligibility will not preclude leveraging prior knowledge across applications submitted by the same sponsor
• Meetings to discuss the possibility of platform technology designation can be requested in accordance with FDA’s Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products Draft Guidance and designation requests themselves should be submitted concurrent with initial submission of, or as an amendment to, an IND
• Platform technology designations may be revoked by FDA if eligibility criteria are no longer met; revocation will be communicated in writing together with FDA’s revocation rationale
• Post approval manufacturing supplements can be used to seek approval for changes to platform technologies; a single submission of grouped supplements can be utilized by a sponsor of multiple approved applications utilizing the platform technology; in advance of a planned change to a designated platform technology, an original application or a prior approval supplement can include one or more comparability protocols to provide for future changes to the platform technology

Finally, FDA notes that potential benefits for sponsors receiving platform technology designation include the following:

• Receiving timely advice and having additional engagement with FDA during product development, where FDA may prioritize interactions where platform technology designation can have the most significant public health benefit or impact
• Leveraging data from prior approved products, such as batch or stability data
• Leveraging nonclinical safety data to streamline nonclinical work for subsequent products
• Considering previous FDA inspectional findings for subsequent marketing applications that relate to the manufacture of a drug or biologic that incorporates or uses the designated platform technology

Interested stakeholders may submit comments to the FDA on the Draft Guidance to Docket FDA-2024-D-1829 until July 29, 2024. Please contact any of the authors or your Goodwin attorney with any questions or if you would like to submit a comment.

This informational piece, which may be considered advertising under the ethical rules of certain jurisdictions, is provided on the understanding that it does not constitute the rendering of legal advice or other professional advice by Goodwin or its lawyers. Prior results do not guarantee a similar outcome.