Placeholder Banner

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(BIO) thanks the Food and Drug Administration (FDA or Agency) for the opportunity to submit comments on the Draft Guidance for Industry “Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations” (Draft Guidance or Guidance).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 BIO generally finds the Draft Guidance helpful and we believe that the framework is drafted in a way that supports development of innovative gene therapy products. BIO appreciates the three bulleted “cases” provided by FDA in the Draft Guidance and believes the “cases” are sufficiently exhaustive in demonstrating under what circumstances FDA would consider two gene therapy products either the same or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.

Download Full Comments Below
Discover More
On Thursday, June 10th, 2021, the U.S. Food and Drug Administration (FDA) published a draft guidance, Core Patient-Reported Clinical Outcomes in Cancer Clinical Trials, providing recommendations to sponsors on the collection of a core set of patient…
On Wednesday, July 14th, BIO submitted comments on FDA’s Guidances: 1) Remote Interactive Evaluations of Drug Manufacturing and Bioresearch Monitoring Facilities During the COVID-19 Public Health Emergency Guidance; 2) Updated Manufacturing, Supply…
On Tuesday, April 6th, BIO submitted comments on the U.S. Food and Drug Administration (FDA)’s January 2021 draft industry guidance on “Human Gene Therapy for Neurodegenerative Diseases.” In its comments, BIO stated that while the guidance covers…