To speed up drug applications, the FDA has laid out new guidance on how to request a platform designation that can smooth subsequent applications for biotechs working off the same reproducible technology.
The agency defines a platform technology as any well-understood reproducible technology, including a nucleic acid sequence, molecular structure, mechanism of action, delivery method, vector or a combination of these.
While the FDA has always allowed drug application sponsors to use prior knowledge of a therapy to support future applications, the agency has not had a formal way to apply that to platforms that can churn out multiple drugs. This is especially key for companies in the gene editing space, where a platform can create multiple treatments for multiple different genes.
The new guidance explains that once a sponsor receives an abbreviated new drug application (ANDA), new drug application (NDA) or biologics license application (BLA) for a treatment, they can then request a platform designation. That will ease future drug applications for subsequent treatments from the same technology.
Biotechs should submit the designation request during the IND phase for the next treatment off the line. At this point, the FDA explains, the sponsor should have enough information to outline how the proposed platform technology would meet the eligibility requirements. The FDA will respond within 90 days.
The guidance was created by the FDA’s Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER).
CBER Director Peter Marks, M.D., Ph.D., hinted at the pending guidance during the American Society of Gene & Cell Therapy conference in Baltimore earlier this month.
“We have to be thinking now because if every base pair changes we're going to have people come back to the agency, there are a lot of nucleotides out there that can go wrong in the genome. And I don't think we can have 3 billion visits to the FDA a year, so we'll have to figure this out,” Marks said during a fireside chat.
He recommended that companies come to the FDA equipped with cell-based analysis to prove there are no off-target effects and detailed manufacturing information. For therapies that target gene mutations, Marks said the FDA wants to see data to support the correction at the right site. If a therapy can target 30 or so gene mutations, the agency would want to see correction at one or two of the sites. If a positive clinical endpoint is achieved then it’s game-on for future mutation requests.
“This is not in stone yet, but what we're thinking about is that you'd probably then just give an approval for all the mutations in that gene so you're not having to have people come back every time,” Marks said.
There are, of course, caveats in the complex world of gene editing, but Marks said the goal is to ease the burden on both the agency and the companies.
Marks encouraged regulatory professionals at gene editing companies to be thoughtful about the initial mutations they choose to take to the agency.
The FDA is accepting comments on the platform designation guidance until July 29.