Home / Health / FDA Declines Regenxbio's Hunter Syndrome Drug
FDA Declines Regenxbio's Hunter Syndrome Drug
10 Feb
Summary
- FDA has not approved Regenxbio's RGX-121 for Hunter syndrome.
- Regenxbio plans to resubmit its marketing application.
- RGX-121 targets the ultra-rare neurodegenerative disease Hunter syndrome.
Regenxbio announced on Monday, February 9, 2026, that the U.S. Food and Drug Administration has declined to approve its drug candidate, RGX-121. This medication was intended for the treatment of Hunter syndrome, a rare and severe neurodegenerative disease.
The biotechnology company stated its intention to work closely with the FDA. Regenxbio aims to address the agency's concerns and resubmit its marketing application. RGX-121 targeted Mucopolysaccharidosis II, the medical term for Hunter syndrome.
This decision represents a setback for Regenxbio's efforts to bring a new therapy to patients suffering from this ultra-rare condition. The company remains committed to pursuing approval for RGX-121.
