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Orphan Drugs: BIO Comments on Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations

June 23, 2020

The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA or Agency) for the opportunity to submit comments on the Draft Guidance for Industry “Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations” (Draft Guidance or Guidance).

BIO appreciates FDA’s efforts to provide drug developers with guidance pertaining to what features FDA will consider when making determinations of “sameness” for gene therapies in the context of orphan drug designation, especially as the landscape for gene therapy products continues to evolve. BIO generally finds the Draft Guidance helpful and we believe that the framework is drafted in a way that supports development of innovative gene therapy products. BIO appreciates the three bulleted “cases” provided by FDA in the Draft Guidance and believes the “cases” are sufficiently exhaustive in demonstrating under what circumstances FDA would consider two gene therapy products either the same or different. However, we do request that FDA clarify that gene editing products are out-of-scope for the guidance and will be subject to case-by-case determinations. 

The complete comment letter can be found below.

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