In May 2024, FDA issued the draft guidance, Platform Technology Designation Program for Drug Development. This guidance is applicable to drug products approved through the New Drug Application (NDA) or Abbreviated New Drug Application (ANDA) routes and biological products approved through the Biological License Application (BLA) route.
I found this guidance very interesting and informative. One of the first questions I receive from clients is, “Is this something that is applicable to my program?” The answer to this question has both a good news and a bad news component.
The first step in understanding this guidance is to understand the definition of a platform technology. The guidance defines it as “a well-understood and reproducible technology, which 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 the sponsor demonstrates that the technology is incorporated in or used by a drug or biological product and is essential to the structure or function of such drug or biologic product; can be adapted for, incorporated into, or used by, more than one drug or biological product sharing common structural elements; and facilitates the manufacture or development of more than one drug or biological product through as standardized production or manufacturing process or processes”.
Tucked back at the end of the guidance, FDA lists a number of examples of potential platform technologies. These include many familiar types of products such as lipid nanoparticle (LNP) platform delivery systems, which have been used for vaccine, gene therapy and oligonucleotide delivery, monoclonal antibodies, and platforms using a targeting moiety in conjunction with a well characterized synthetic short interfering (si)RNA. There are a number of these products on the market and many are very familiar to us.
So what is the bad news?
In order to be eligible for platform technology designation, the first product using the particular platform needs to have been approved.
It is with subsequent products that the request for platform technology designation is applicable. This designation may speed up the development of the next product using the same technology. Data can be leveraged from the prior product development and may include manufacturing specifications and information, non-clinical safety data, and prior FDA inspection findings.
One can only ask for platform technology designation if one is the Sponsor of the initial (approved) product and the developmental product or if one has full right of reference to the initial product. This is very important as one cannot automatically leverage information from another Sponsor.
The requirement for platform technology designation is that it is incorporated in or used by an approved drug and preliminary evidence demonstrates that 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. Data or information submitted in the designation request must indicate that the platform technology has a reasonable likelihood to bring significant efficiencies to the drug development or manufacturing process and to the FDA review process.
Preliminary evidence is defined as completed tests or studies comparing the platform technology used in the approved product with its proposed use in the drug under investigation.
A designation request should include all products using the technology regardless of the stage of development.
Requests for platform technology designation are appropriate during the IND stage and the determination by FDA is within 90 days of receipt of the request. While a request can be submitted at the same time as the initial IND submission, because of the requirement for data on the product under investigation, it is generally more appropriate after IND filing, after additional data satisfying FDA’s requirements have been generated.
The benefits of platform technology designation include early interactions with FDA, timely FDA advice, and additional FDA engagement during the drug development process. Of note, fast track, breakthrough therapy designation, and priority review are not automatically included if one gains platform designation status, but need to be applied for and approved separately at the appropriate stage of development.
