FDA Commissioner Releases Statement on Gene Therapies

Agency recognizes growth of gene therapy as an option

FDA Commissioner Scott Gottlieb, MD, released a statement last week to acknowledge and address the advance of gene therapies as an option in the United States.

“Once just a theory, gene therapies are now a therapeutic reality for some patients,” the statement began. “These platforms may have the potential to treat and cure some of our most intractable and vexing diseases.”

Dr. Gottlieb went on to suggest the framing of policy will be the determining factor for further advancement, encouraging the fullest development for the purposes of treating conditions that include genetic disorders, autoimmune diseases, heart disease, cancer and HIV/AIDS.

“We know that we still have much to learn about how these products work, how to administer them safely, and whether they will continue to work properly in the body without causing adverse side effects over long periods of time,” the statement continued. “In contrast to traditional drug review, some of the more challenging questions when it comes to gene therapy relate to product manufacturing and quality, or questions about the durability of response, which often can’t be fully answered in any reasonably sized pre-market trial.”

SOURCE: Food and Drug Administration

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