Following the FDA’s May release of long-awaited draft guidance explaining how companies can win designations for certain cell and gene therapy “platform” technologies, companies like Regeneron, Alnylam, Verve Therapeutics and others are seeking more clarity from the agency on the program.
Nearly all of the recently released comments praised the FDA for creating this new designation, which can potentially expedite new technologies, processes and products to treat complex diseases. But many companies and government agencies are seeking more clarity and transparency on how the designation, which would label some cell and gene technologies as “platforms” for use across multiple diseases, will function in the real world.
Regeneron, for instance, called on the agency to work more closely with other regulators, particularly in the EU, on honing the process of designating the technologies. The New York-based company says it’s still unclear on whether multiple platform designations can be applied for the same product.
“This would benefit complex platforms such as gene insertion therapies where two or more components could qualify as distinct platforms, and the formal designation for the technologies independently could yield manufacturing, development, and review efficiencies,” Regeneron head of regulatory intelligence Kellie Taylor writes.
Verve notes that in the examples of key elements of a potential platform technology, FDA identifies what would be consistent depending on the modality, but still needs to go a step further.
“It is not clear whether the examples provided are FDA expectations or whether distinct key elements might be held consistent to compromise a platform technology,” Verve’s executive director of regulatory strategy Alex Bush said.
Alnylam also asked for more clarity from FDA on when to submit the designation requests since such a request must follow at least one product being approved after already using the platform technology.
The US Biomedical Advanced Research and Development Authority (BARDA) similarly needed more clarity on when to make such requests, and asked the FDA if this “designation process need[s] to be repeated for each new IND/BLA” and how this designation might work if a product has been authorized under the EUA pathway.
Industry group BIO similarly called on FDA to clarify in its final guidance how the agency plans to apply its guidance consistently across review divisions and between CDER and CBER.
“For example, the guidance acknowledges that prior knowledge and platform approaches historically have been, and can continue to be, leveraged, even where a program may not qualify for PTD [platform technology designation]. How this works in practice, however, is not transparent,” said Emily Acker, director of infectious disease policy at BIO.
While the draft guidance notes the possibility for sponsors of new drug applications leveraging platform tech from other applications submitted by the same sponsor, the US Department of Defense questions whether FDA will ultimately create a published list of platform technology designations with data.
Most companies holding the related information and data of the platform will want it to remain confidential, the Defense Department wrote. And when using contract manufacturers, “without a general registry, it will be impossible to know what is out there. The guidance is not clear about this,” the department said.
CDMO WuXi Biologics, under pressure from Capitol Hill from the Biosecure Act, also questioned the FDA on what happens if the platform technology belongs to a contractor: “What would be the communication and submission pathways for CMO and CDMO?”