> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# No REMS Needed for Gene Therapy: FDA
- URL: https://www.fdaweb.com/no-rems-needed-for-gene-therapy-fda/
- Published: 2022-11-28T12:00:00.000Z
- Updated: 2026-09-14T18:07:37.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5153270

In approving 11/22 Behring’s Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy to treat adults with Hemophilia B (congenital Factor IX deficiency), FDA simultaneously denied a citizen petition from the National Hemophilia Foundation (NHF) that sought restrictive Risk Evaluation and Mitigation Strategies (REMS) for the drug.

In [denying the petition](https://downloads.regulations.gov/FDA-2022-P-1444-0006/attachment%5F1.pdf?ref=fdaweb.com), FDA said etranacogene dezaparvovec’s approved labeling adequately informs prescribers and patients of the risks associated with its use. The NHF petition stated that “\[s\]teroid use should be an outcome of interest, given ... the high proportion of related adverse events,” However, the agency said that its review of the clinical studies did not show any significant reports of thrombosis or adverse events related to steroid use.

Additionally, the petition requested that FDA “\[i\]nclude the eligibility (inclusion and exclusion) criteria utilized in the clinical trials on the drug label.” The agency said, however, that NHF had not provided information showing that including the complete eligibility criteria used in the clinical trials in the drug labeling “would provide meaningful information regarding how to use the drug safely and effectively and, based on our review, FDA is not aware of any reason why its inclusion would do so.”