FDA clinical hold for Regenxbio’s RGX-121 for MPS

25 August 2026

Shares of Regenxbio (Nasdaq: RGNX) plummeted 25% to $8.05 following the US Food and Drug Administration’s (FDA) clinical hold on gene therapy candidate RGX-121 (clemidsogene lanparvovec), for the treatment of Mucopolysaccharidosis type II (MPS II).

The FDA placed a clinical hold on RGX-121 following the discovery of asymptomatic spine MRI findings in five participants in the CAMPSIITE study; Regenxbio says it does not expect to resubmit the RGX-121 Biologics License Application (BLA) in the near term.

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