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FDA Braces for Looming Boom in Cell and Gene Therapy Submissions

June 12, 2023

Ana Mulero, BioSpace

With contributions from:
Greg Meyer, Vice President, Regulatory Affairs, Premier Consulting
Kenneth Ndugga-Kabuye, Vice President, Gene and Cell Therapy, Premier Research

Over the past few years, the pipeline for cell and gene therapies has been increasing at a rapid pace, and the FDA is now ready, thanks to the establishment of a new, specialized super office.

“There could be around 10 total regulatory decisions this year on cell and gene therapies, including up to five decisions on gene therapies for rare genetic diseases alone,” Stephen Majors, global head of communications at the Alliance for Regenerative Medicine (ARM), told BioSpace.

These could include several potential firsts, including the first CRISPR therapy—Vertex Pharmaceuticals and CRISPR Therapeutics’ exa-cel for the treatment of sickle cell disease; and the first gene therapy to treat Duchenne muscular dystrophy (DMD)—Sarepta Therapeutics’ SRP-9001, which could become the second gene therapy to receive approval via the agency’s accelerated approval pathway.

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