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Public Policy Newsletter Article

Biocom Submits Comments on Interpreting the Sameness of Gene Therapy Products

  • 2020-07-30T15:00:00.000+0000
  • Washington DC
  • Author: Brittany Blocker

On July 20, Biocom submitted comments in response to the Food and Drug Administration’s (FDA) draft guidance, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations. The draft guidance explains how the FDA will decide if orphan exclusivity will be awarded if two gene therapy products are intended for the same use or indication. To grant orphan-drug designation and determine eligibility for orphan-drug exclusivity, assuming that two gene therapy products are intended for the same use or indication, the FDA’s determination of “sameness” will consider the principal molecular structural features of the gene therapy products.

For gene therapy products, the draft guidance, states the FDA’s determination of sameness will be based on the principal molecular structural features such as transgenes and vectors. The Agency provides case examples to demonstrate scenarios where it would consider two gene therapies as the same or different. FDA will consider that two gene therapy products are different if they express different transgenes and/or use different vectors. If vectors from the same viral class are used then sameness will be determined on a case-by-case basis. When applicable, the FDA also generally intends to consider additional features (e.g., regulatory elements, cell type that is transduced) that can contribute to the therapeutic effect.

Biocom is generally supportive of the draft guidance and recommends the FDA to clarify what constitutes a “minor difference” or “additional feature” and provide examples of how determinations are made in those cases.