As Ori Wins Designation, FDA’s Advanced Manufacturing Program Nears Proof Point

Last week, Ori Biotech announced that it has joined the short list of companies to receive advanced manufacturing technology designation from the FDA. Seeking to support uptake of new manufacturing technologies, the agency has said AMT will involve early, priority interactions with drug developers using designated technologies. The scheme has attracted industry interest, and nine months after finalizing the initiative, the FDA is on the cusp of showing whether the designation can deliver on its objectives.

Absent an AMT designation, each sponsor that uses a technology needs to submit comprehensive manufacturing information as part of their investigational new drug (IND) and drug approval applications despite making products in a similar way to every other user of the platform. According to the FDA, the agency established the AMT designation to streamline this process, encouraging early adoption of technologies that improve the reliability and robustness of manufacturing processes, enhance product quality, reduce drug development time or increase or maintain the supply of certain critical drugs.

To date, Ori is one of three companies that have announced publicly that they’ve received AMT designation. Cellares received AMT designation in April for technology for automating cell therapy production. Cellino secured a designation for its induced pluripotent stem cell manufacturing platform in May. Ori’s designation is for its IRO system for automating, digitizing and standardizing parts of the cell and gene therapy manufacturing process.

Ori CEO Jason Foster told BioSpace the company collated data from 10 to 12 customers to show the FDA that its system is robust and can improve consistency while increasing throughput. Foster said the agency submitted two rounds of questions seeking specifics on the broader use of the platform, and that the designation process would be very familiar to companies that have had other interactions with the agency.

“Now the fun work starts,” Foster said. “The crucial piece is how do we leverage AMT to help our partners get products into the clinic more quickly and get them to patients more quickly?”

Foster said the stated benefits of the designation, such as priority interactions with the FDA, should help Ori’s clients accelerate IND submissions and reach the clinic faster. The Ori CEO also expects clients to have “an easier time” when discussing chemistry, manufacturing and controls (CMC) with the agency.

“We’re submitting a master file that will have a lot of this [CMC] data in it so that they can reference that and reference the IRO technology as the AMT-designated technology, so that those boxes are ticked,” Foster said. “A lot of the regulatory conversations about cell therapies are around manufacturing, so the ability to streamline that discussion would be well accepted by our partners and by the agency.”

Michael Paglia, chief technology officer at ElevateBio BaseCamp, told BioSpace the validation that comes from AMT designation could help technology providers overcome a barrier to the use of their products. At ElevateBio, Paglia and his colleagues use proprietary and partnered technologies to develop and make cell and gene therapies for clients. ElevateBio is part of Ori’s preferred partner network.

Paglia joined ElevateBio before its official launch in 2019 and now evaluates new manufacturing technologies from other companies. The experience has shown him what evidence companies want to see when deciding whether to work with a new service provider or adopt a novel technology, he said.

“The challenge is, ‘Okay, you built this beautiful facility, you have great, talented people, but how many batches have you made clinically? How many INDs have you supported? Has your lentiviral vector been in a clinical or a commercial product? Do you have an approved product that you’ve manufactured for? Have you been part of a BLA?’” Paglia said, recounting the types of questions he often hears from drug developers.

Paglia said the technology developers that he evaluates do a great job on tasks such as toxicology studies and putting together device master files. However, the critical question for ElevateBio’s clients is whether a technology has been used to make a therapy for a patient. Paglia said AMT designations could give companies developing new technologies “a seal of approval, so to speak, from the FDA.”

Foster expects the first Ori partners using the AMT designation to support filings to have candidates in clinical trials in the next six months. The filings will begin to show whether the IRO platform and AMT designation can significantly shorten R&D programs that often take 10 years or more.

“We think we can shave about three years off the development timeline with a combination of faster tech transfer, faster optimization, but also hopefully this prioritization of review through the FDA,” Foster said, adding that that would save “a huge amount of money, a huge amount of time.”

Foster sees these savings as critical to the viability of cell and gene therapies, some of which have improved clinical outcomes but underwhelmed commercially. Reducing the cost of developing and making cell and gene therapies, including by lowering the rate of manufacturing failures, could address one barrier to uptake by driving down the price of the treatments, he said.

If the AMT designation is to contribute to those improvements, the FDA will need to remain committed to the idea amid changes at the agency. The designation emerged during Peter Marks’ time as director of the Center for Biologics Evaluation and Research. Marks left the agency in March. But Foster cited the views voiced by members of the new regime at an event in June as evidence of FDA’s ongoing support for advanced therapies.

“Every indication we have,” the Ori CEO said, “is that the administration continues to greatly support advanced therapies and wants to open up access for patients, which would mean further supporting the AMT designation.”