FDA Policy/General

FDA Pushes Back Review of Hunter Syndrome BLA

FDA has extended by 90 days its review of a Regenxbio’s BLA for RGX-121 (clemidsogene lanparvovec), an investigational gene therapy for Hunter syndrome, a rare and life-threatening genetic disorder. The agency’s extension pushes back the user fee review action target date from 11/9 to 2/8/2026.

RGX-121 is designed as a one-time treatment to deliver a functional copy of the gene responsible for producing the enzyme iduronate-2-sulfatase (I2S), according to the company. If approved, it would be the first therapy intended to directly target the underlying cause of Hunter syndrome, which currently has no options to address the neurodevelopmental decline associated with the disease, it says.

The extension follows Regenxbio’s submission of longer-term data from its pivotal study of 13 boys with Hunter syndrome. The 12-month results, the company says, were consistent with earlier findings showing improvements in biomarkers and neurodevelopment. These data will be presented at the International Congress of Inborn Errors of Metabolism next month.

Regulatory reviews of the therapy remain on track, the company notes, adding that FDA recently completed both a pre-license inspection and a bioresearch monitoring inspection with no observations, and no safety concerns raised to date.

Hunter syndrome, or mucopolysaccharidosis Type II, is an X-linked recessive disorder caused by I2S deficiency, which leads to the buildup of toxic molecules in tissues and organs, according to Regenxbio. It says severe forms of the disease cause developmental delays by early childhood, with neurological symptoms representing a major unmet medical need.

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FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome