FDA’s gene therapy discussions stump industry experts on specifics of upcoming guidance for early scalable manufacturing; optimism perseveres on agency's tone

25 May 2018

Gene therapy developers face intricate manufacturing scale-up challenges that will likely take years to resolve, despite the FDA’s intention to issue guidance on solidifying CMC commercial strategies at the IND stage, industry executives noted.

The close knit alliance of developers are, however, optimistic the FDA is prioritizing regenerative medicine, executives told this news service on the sidelines of this week’s Alliance for Regenerative Medicine (ARM) meeting in Washington, DC. They noted the agency is acknowledging gene therapies have clear efficacy, but the manufacturing piece needs fine-tuning to help expedite approvals on smaller studies.

Gene therapy manufacturing expertise will hold significant weight, experts said, with some noting the CMO industry spotlight may trigger more partnerships and acquisitions. Yet, drug developers are also likely to invest in internal manufacturing capabilities, several experts added.

FDA’s draft guidance is going through final clearance and will be published in the next couple of months, followed by an open comment period, said Scott Gottlieb, the agency’s commissioner, during his speech at the meeting. Gene therapy trials may only need a few dozen individuals, and the FDA wants to provide the CMC must-haves and encourage sponsors to scale their manufacturing capabilities early, he added.

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