REGENXBIO, Inc. – Complete Response Letter (BL 125840)

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g YN U.S. FOOD & DRUG ADMINISTRATION Our STN: BL 125840/0 COMPLETE RESPONSE February 7, 2026 REGENXBIO, Inc. Attention: Steve Pakola, MD 9804 Medical Center Drive Rockville, MD 20850 Dear Dr. Pakola: Please refer to your Biologics License Application (1b) (4) , for clemidsogene lanparvovee, (1) We have completed our review of all the submissions you have made relating to this BLA. After our complete review, we have concluded that we cannot grant final approval because of the deficiencies outlined below. You submitted a BLA for clemidsogene lanparvovec, a gene therapy for the requested indication of treatment of patients with mucopolysaccharidosis type II (MPS II, Hunter syndrome). Your requested broad indication implies both neuronopathic and attenuated (non-neuronopathic) types of MPS II. MPS Il is a rare disorder of metabolism caused by mutations in the iduronate-2- sulfatase (IDS) gene. Deficiency of lysosomal enzyme results in progressive accumulation of glycosaminoglycans (GAGs) heparan sulfate (HS) and dermatan sulfate (DS) throughout body tissues and organs, leading to multi-organ dysfunction. The neuronopathic form of disease is distinguished from the attenuated form by presence of neurological impairment. To support this BLA under the accelerated approval pathway, you submitted the results from a single study, RGX-121-101, an open-label, multi-part study; Part 1 focused on dose-finding and safety, and Part 2 served as the pivotal single-arm study which eval

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