FDA rejects Regenxbio’s MPS II rare-disease gene therapy

Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54.

The Food and Drug Administration has rejected a rare-disease gene therapy from Regenxbio, the company said Monday. 

The one-time treatment, called RGX-121, is designed to replace a malfunctioning gene that causes mucopolysaccharidosis type II, also known as Hunter syndrome, an ultra-rare disorder that causes physical and cognitive impairments. 

Regenxbio had applied for accelerated approval, a type of conditional market clearance, based on RGX-121’s ability to significantly reduce in the short term a specific biomarker in cerebrospinal fluid believed to correlate with longer-term cognitive improvements in patients with the severe form of Hunter syndrome. 

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