The FDA has placed a clinical hold on REGENXBIO's RGX-121 gene therapy for Hunter Syndrome due to asymptomatic spine MRI findings in study participants, delaying its Biologics License Application. This significantly impacts the company's near-term pipeline and revenue projections for this specific therapy.
REGENXBIO announced that the FDA has placed a clinical hold on its investigational gene therapy, RGX-121, for Mucopolysaccharidosis type II (Hunter Syndrome). This decision follows the discovery of asymptomatic spine MRI findings in five participants in the CAMPSIITE study, leading the company to not expect a near-term resubmission of the Biologics License Application (BLA). This is a significant setback for RGNX, as RGX-121 was a key pipeline asset, and the delay will impact its potential market entry and future revenue streams. While the company states these findings are likely benign and unique to this program, and they will focus on other candidates, the immediate impact is negative for RGNX's stock due to increased uncertainty and delayed commercialization for RGX-121.