Drugs

FDA Issues Gene Therapy Guidances

FDA has issued six final guidances on gene therapy manufacturing and clinical development of products plus a draft guidance on interpreting the sameness of gene therapy products under the orphan drug regulations. In an agency statement (contains links to the guidances), CBER director Peter Marks says “as regulators of these novel therapies, we know that the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field and further enable innovators to safely develop effective therapies for many diseases with unmet medical needs.”

The statement says the six final guidances provide FDA recommendations for product developers on manufacturing issues and recommendations for those focusing on gene therapy products to address specific disease areas. The documents incorporate input from many stakeholders and take a significant step toward helping to shape the modern structure for the development and manufacture of gene therapies, the statement says.

The draft guidance on interpreting sameness of gene therapy provides the agency’s proposed current thinking on an interpretation of sameness between gene therapy products for the purpose of obtaining orphan drug designation and eligibility for orphan drug exclusivity. The draft is said to focus on how FDA will evaluate differences between gene therapy products that are intended to treat the same disease. The document says the agency will consider the principal molecular structural features of the gene therapy products, including transgenes and vectors.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome