> ## 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.

# Hemophilia Group Wants REMS for 2 Investigational Gene Drugs
- URL: https://www.fdaweb.com/hemophilia-group-wants-rems-for-2-investigational-gene-drugs/
- Published: 2022-07-11T12:00:00.000Z
- Updated: 2026-09-14T17:51:10.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5152244

The National Hemophilia Foundation says FDA should require Risk Evaluation and Mitigation Strategies (REMS) if it approves two investigational gene therapies to treat hemophilia. The foundation’s 7/1 [petition](https://downloads.regulations.gov/FDA-2022-P-1444-0001/attachment%5F1.pdf?ref=fdaweb.com) says REMS should be required for BioMarin’s valotocogene roxaparvovec, under regulatory review to treat severe hemophilia A, and CSL Behring’s etranacogene dezaparvovec, under review to treat hemophilia B.

The petition says the agency should require as part of a REMS elements to assure safe use that includes:

- training and education for doctors and healthcare providers on gene therapy and the management of people with hemophilia who receive a gene therapy product;
- training and education on shared decision-making for doctors and healthcare providers who will evaluate, administer, and follow people with hemophilia who are candidates to receive a gene therapy product;
- certification of facilities administering the two investigational gene therapies;
- a requirement that the two therapies be administered only at a federally recognized hemophilia treatment center with knowledge and expertise in evaluating, administering, and managing people with hemophilia who have received investigational gene therapy products; and
- enrollment of individuals receiving the gene therapy products in a registry to collect robust data, including on adverse events of interest.