Regenxbio’s BLA for RGX-121 in Hunter syndrome accepted by FDA

Regenxbio (RGNX) announced the FDA has accepted for review the Biologics License Application, or BLA, seeking accelerated approval for clemidsogene lanparvovec, or RGX-121, for the treatment of Mucopolysaccharidosis II, or MPS II, also known as Hunter syndrome. The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act target action date of November 9. RGX-121 has received Orphan Drug Product, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy designations from the FDA and advanced therapy medicinal products classification from the European Medicines Agency. Under the strategic partnership announced in January 2025, following potential FDA approval, RGX-121 will be commercialized by NS Pharma, a wholly-owned subsidiary of Nippon Shinyaku (NPNKF), in the U.S. Approval of RGX-121 could result in receipt of a Priority Review Voucher. Regenxbio retains all rights to, and 100% of any proceeds related to the potential sale of, the PRV for RGX-121.