REGENXBIO Inc. announced new, positive 12-month data from the Phase I/II/III CAMPSIITE trial of clemidsogene lanparvovec (RGX-121) for Mucopolysaccharidosis Type II (MPS II) on September 5, 2025. The data was presented at the International Congress of Inborn Errors of Metabolism (ICIEM) 2025.
Pivotal participants sustained an 82% median reduction of cerebrospinal fluid (CSF) heparan sulfate (HS) D2S6 levels at one year, a key biomarker of MPS II brain disease. Positive neurodevelopmental outcomes were observed, and a strong correlation was found between CSF HS D2S6 levels and neurocognitive outcomes.
These longer-term data were submitted to the FDA in response to an information request for the ongoing Biologics License Application (BLA) review of RGX-121. The FDA completed inspections with no observations and no safety concerns, with a decision expected by February 8, 2026.
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