REGENXBIO, Inc. · 完整回复函 (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

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