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BioCommunique Article

​​​​​Agency released final guidance documents on development of gene therapies for specific disease categories: hemophilia, retinal disorders, and rare diseases

FDA Finalizes Six Gene Therapy Guidances; Releases New Draft Guidance

  • 2020-01-31T17:19:00.000+0000
  • Washington DC
  • Author: Brittany Blocker

On January 28, the Food and Drug Administration finalized six guidance documents on gene therapy development and issued a draft guidance on interpreting the sameness of gene therapies under the orphan drug regulations.

The agency released final guidance documents on the development of gene therapies for specific disease categories: hemophilia, retinal disorders, and rare diseases. The FDA also finalized updates to three existing guidance on chemistry, manufacturing, and control (CMC) for investigational drug applications, testing of retrovirus during product manufacture and patient follow-up, and long-term follow-up after the administration of human gene therapy products. These updates will replace the guidances issued in 2006 and 2008.

The draft guidance on interpreting sameness explains how the FDA will decide if orphan exclusivity will be awarded if two gene therapy products are intended for the same use or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. For the purpose of granting orphan-drug designation and determining 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. See FDA’s announcement here.

Biocom is requesting member feedback on the draft guidance. As of now, a comment deadline has not been established. We will provide an update with timelines when we have additional details.