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# Regenxbio to File BLA for Hunter Syndrome Treatment
- URL: https://www.fdaweb.com/regenxbio-to-file-bla-for-hunter-syndrome-treatment/
- Published: 2022-08-04T12:00:00.000Z
- Updated: 2026-09-14T17:54:51.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5152442

Regenxbio says it will file a BLA with FDA using the accelerated approval pathway for its RGX-121 to treat mucopolysaccharidosis type 2, also known as Hunter Syndrome. The company says its pivotal trial for RGX-121 is active and enrolling patients. Regenxbio describes RGX-121 as an investigational, one-time AAV (adeno-associated virus) therapeutic using the NAV AAV9 vector to deliver the gene that encodes the iduronate-2-sulfatase enzyme.

Company officials say their most recent discussions with FDA supported the plan to submit a BLA through the accelerated approval pathway.