Update: FDA-Approved Cell & Gene Therapies

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When the FDA gave Novartis the historic green light for Kymriah – the first gene therapy approved in the United States – in 2017, the agency declared that it was “ushering in a new approach” to the treatment of cancer and other serious and life-threatening diseases. Today, more than 20 cell & gene products have been approved. Here is an update of the products with a license from the FDA.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said then-commissioner Scott Gottlieb at the time of the Kymriah announcement.

Five years later, the number of cellular and gene therapy products approved by the FDA has jumped to 23, including one for spinal muscular atrophy (also from Novartis), and others for cartilage defects of the knee, retinal dystrophy, several types of cancer, and thermal burns.

The licenses are given by the regulator’s Office of Tissues and Advanced Therapies (OTAT).

The FDA’s current guidance on expedited programs available to sponsors of regenerative medicine therapies include traditional Fast Track and Breakthrough Therapy designations and Regenerative Medicine Advanced Therapy Designation (RMAT), which was created by the 2016 “21st Century Cures Act.”

In conversation with PharmaBoardroom back in 2020, Dr Peter Marks, the director of the FDA’s Center for Biologics Evaluation and Research (CBER) – the department in charge of OTAT – explained the difference and advantages of RMAT designation.

“The RMAT designation is very much like the Breakthrough Therapy designation but with certain features targeted for regenerative medicine products… [for RMAT] the company simply has to show evidence of activity against the disease in question, instead of showing that the therapy is better than a standard of care,” he said.

Dr Marks also outlined the FDA’s ambition to be the world’s gene therapy leader: “We are trying to take a fair amount of leadership in gene therapies. We are very interested in helping to move the field forward by looking at how one can develop gene therapies for small patient populations and also how to do that in what would ultimately be a commercially viable manner,” Dr Marks said.

“We know the statistics of product development: only about 10 percent of the therapies that make it into Phase 1 trials will ever see the light of day. Cell therapies are not all that different at this point. I am sure we will see some in the near future but it is taking a little longer than might have been thought.”

Here is an updated list of licensed products from the FDA’s Office of Tissues and Advanced Therapies (OTAT):


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