Cell and gene therapy experts question where the FDA designation fits in an environment that features a range of intersecting regulatory perks.
The FDA’s recently created advanced manufacturing technology designation has a clear goal: Facilitating drug development. However, as the program approaches its first birthday, questions remain about how the chemistry, manufacturing and controls (CMC)–focused designation will interact with other regulatory statuses and what it will mean for companies engaging with the agency.
Congress mandated the creation of the AMT designation in the Food and Drug Omnibus Reform Act of 2022. The designation was devised for novel technologies that substantially improve the manufacturing process, including by reducing development time and supporting the production of certain drugs affected by supply disruptions. Lawmakers told the FDA to expedite filings supported by AMT statuses and to allow applicants to reference or rely on the designations in their submissions.
The FDA shared more information about the designation in final guidance published late last year. Officials said key benefits include early interactions regarding the development and manufacture of drugs using a designated AMT. According to the guidance, the FDA intends to provide timely advice, engage in additional communication and prioritize applicant interactions involving the use of a designated AMT.
Those benefits appear to overlap with the advantages provided by other regulatory statuses available to cell and gene therapy manufacturers, such as the Regenerative Medicine Advanced Therapy designation. The qualifying criteria for fast track, breakthrough and regenerative medicine designations differ, but the level of overlap between the evidence requirements was a source of confusion for the industry when the FDA first proposed RMAT. Companies progressively collect the evidence needed to receive each designation. For example, uniQure’s Huntington’s disease gene therapy received fast track status in 2019, RMAT in 2024 and breakthrough designation in 2025.
The AMT qualifying criteria are distinct in that they are focused on manufacturing rather than efficacy, but insiders have observed that potential users of the designation may have the preclinical or clinical data to secure other statuses. Michael Paglia, chief technology officer at ElevateBio BaseCamp, told BioSpace that many cell and gene therapy companies that he works with already have designations that allow early interactions with the FDA.
Paglia and other experts have ideas about what AMT designations may mean to companies that already have expedited access to the FDA. But with only three companies having publicly announced their receipt of an AMT designation, and with drug developers yet to put the benefits of the new program to use, the details remain uncertain.
Resolving the Uncertainty
Companies with multiple designations may be able to interact more with the agency by setting up meetings focused on specific areas, Paglia said. While noting that he has yet to see how AMT designations will work in practice, Paglia sketched out a theory for how companies can use multiple designations to discuss narrow sets of questions with specific FDA representatives.
“What we’re guessing is that you have the ability now to really focus RMAT-type interactions on clinical, preclinical, [biologics license applications], commercialization—and then the AMT on more the CMC side of things,” Paglia said.
However, Don Fink, a regulatory expert at Dark Horse Consulting, has doubts about whether AMT designations will open up more access to the FDA. For companies already on the expedited pathways, he told BioSpace, “I don’t think it makes that big a difference, personally, because you’re not going to get a new category of meetings.”
The consultant and former FDA employee outlined hypothetical ways that AMT designations could affect access. For example, if two companies with RMATs request meetings at the same time and only one has an AMT designation, does the FDA prioritize the company with the AMT? Fink does not know the answer yet.
He also speculated about how the FDA would prioritize requests for engagement when one company has an RMAT and another company has an AMT. The consultant predicted that RMAT designation will take precedence, explaining that “once you get one of these expedited designations, you pretty much have your way with the agency for meetings.” The FDA has said an AMT designation does not affect the time needed to review approval filings. By contrast, candidates with product, fast track, breakthrough therapy or RMAT designation may be eligible for expedited review.
While those scenarios suggest AMT designations may have limited impact, Fink speculated that when the FDA is reluctant to grant a Type D meeting, which provides timely feedback critical to move the program forward, the agency may feel obligated to accept the request if the applicant has an AMT designation and questions related to CMC.
A New Card in the Deck
The FDA will now sort through how to apply AMTs on a center-by-center basis, Fink said, and there may be differences in how the designation is used across the agency. A clearer picture may start to emerge in the coming months. Ori Biotech CEO Jason Foster recently said he expects the company’s partners to start using its recently granted AMT designation to support clinical trial filings in the next six months.
There remains the possibility that the designation will be most impactful as a promotional tool for technology providers. Paglia said the designation could be “great for marketing.” Similarly, Fink noted the potential for the status, alongside similar designations, to help companies attract customers and investment.
“Some people just collect them like playing cards. They get a fast track because they can. That’s easy. Then they’ll get an RMAT. Then they’ll get breakthrough [designation], if they can even go further with their clinical data,” Fink said. “They all essentially allow for the same things.”
Fink foresees a possible future in which the designation has little impact beyond those benefits. “Three to five years out it may be just another thing that you have, a shiny nickel coin that doesn’t really make one difference,” he said.
