To date, the U.S. Food and Drug Administration (FDA) has approved four gene therapy products, which insert new genetic material into a patient’s cells. The agency expects a boom in gene therapy product approvals in the coming decade as evidenced by the more than 900 investigational new drug (IND) applications for ongoing clinical studies in this area. The FDA recently issued a press release announcing they believe this will provide patients and providers with increased therapeutic choices. Consequently, the agency announced the release of a number of important policies including: six final guidelines on gene therapy manufacturing and clinical development of products and a draft guidance, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.
Overview: Guidance for Gene Therapy Manufacturers
The six final guidance issued today provide the agency’s recommendations for product developers on manufacturing issues and recommendations for those focusing on gene therapy products to address specific disease areas. The six guidance documents incorporate input from many stakeholders and take a significant step toward helping to shape the modern structure...
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