FDA issues platform technology designation draft guidance

The US Food and Drug Administration (FDA) has published a much-awaited draft guidance detailing how it plans to implement its new platform technology designation program. Drug sponsors can use platform technologies to manufacture more than one drug or biological product through a standardized production or manufacturing process, and the program is intended to provide predictability on how they are reviewed.

The FDA had already allowed the technology when it approved Vertex Pharmaceuticals and CRISPR Therapeutics Casgevy, a gene editing treatment for sickle cell disease, late last year. However, under section 506K of the Federal Food, Drug, and Cosmetic Act (FD&C Act), which was updated as part of the 2022 Prepare for and Respond to Existing Viruses, Emerging New Threats (PREVENT) Pandemics Act, the agency was required to publish a draft guidance by the start of the year. The agency said program should lead to efficiencies when reviewing drugs and biological products that use designated platform technologies.

“This guidance outlines eligibility factors for receiving a platform technology designation, potential benefits of receiving a designation, how to leverage data from designated platform technologies, how to discuss a planned designation request as part of a milestone meeting, the recommended content of a designation request submission, and the review timelines for a designation request,” said FDA in their 28 May announcement of the draft guidance. “This program is intended to result in efficiencies in drug development, manufacturing, and review processes for drug product applications that incorporate designated platform technologies.”

A top FDA official earlier this year noted that while the agency already has some experience with platform technologies, the objective of the guidance is to add more predictability about how it intends to regulate such products. (RELATED: Pharma industry questions FDA on platform technology designation program, Regulatory Focus 23 January 2024)

In the guidance, the FDA notes that in the past, it may have used the term “platform technology,” which doesn’t meet the definition under the statute and the guidance itself, and those technologies would not qualify for the program. The agency, however, also noted that ineligibility for designation under the program doesn’t preclude their sponsors from using the knowledge gained for future drug applications.

FDA said that based on statute, a platform technology is considered a well-understood and reproducible technology that may include nucleic acid sequence, molecular structure, mechanism of action, delivery method, vector, or a combination of such technologies. Sponsors, however, must prove that the technology is essential to the structure or function of a drug or biological product, can be used to manufacture more than one drug or biological product that shares common structural elements, and can be used as a standardized production or manufacturing process to make multiple drugs or biological products.

“This guidance describes those categories and provides recommendations for the types of platform technologies that may be eligible for consideration for designation,” said FDA. “This guidance also gives recommendations for what should be included in submission requests to designate a platform technology, how to update a designated platform technology and, when appropriate, the process for revoking a designation.”

Recognizing a platform technology in the statute entails that the preliminary evidence on the technology demonstrates that it can be used by more than one drug or biological product that doesn’t adversely affect its quality, manufacturing, or safety. The agency defines preliminary evidence as the complete information collected from tests or studies that compare platform technology that has been used for approving or licensing products to the technology used for the investigational drug described in the designation request. Such evidence could include information on structurally similar drug substances, minimal differences in drug formulations, and nearly identical manufacturing processes for drugs or drug product purification.

FDA added that if it has approved an Abbreviated New Drug Application (ANDA), New Drug Application (NDA), or Biologics License Application (BLA) for a drug that incorporates or uses a platform technology, sponsors of subsequent Investigational New Drugs (IND), NDAs, BLAs can request designation of that platform technology to leverage it for new or future applications.

“FDA recommends requesting the designation of a platform technology during the IND phase of drug development for a planned subsequent NDA or 351(a) BLA, because by this stage of development, the sponsor should have sufficient knowledge to outline for FDA how the proposed platform technology would meet the eligibility factors as outlined in section 506K(b) of the FD&C Act,” the agency added. “This should facilitate a more complete request and its timely review by FDA.”

FDA said sponsors can request the designation of a platform technology at any time concurrent with or after they submit an IND, and it should conform to electronic submission guidelines.

"Although a sponsor can request designation concurrent with the submission of an IND, the timing of the request for designation should consider whether there are adequate product-specific data," the agency added. "FDA recommends the sponsor submit far enough into their development cycle to permit a determination of suitability for platform technology designation (e.g., of whether the platform technology has the potential to be incorporated in, or used by, more than one drug without an adverse effect on quality, manufacturing, or safety)."

Reviewers will issue a determination within 90 days of receiving a platform technology designation request, according to FDA.

Stakeholders can comment on the draft guidance until 28 July under docket no. FDA-2024-D-1829 on www.regulations.gov.

Draft guidance

FDA issues platform technology designation draft guidance

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