REGENXBIO, MPS II에 대한 RGX-121 BLA에 대해 FDA CRL 수령

FDA issued a Complete Response Letter on February 7, 2026 rejecting the RGX-121 BLA under accelerated approval. The agency cited uncertainty around neuronopathic patient eligibility, external control comparability, and CSF HS D2S6 as a surrogate endpoint. REGENXBIO plans a Type A meeting and intends to resubmit the BLA with additional data.

핵심 사실

제출: 2026년 2월 10일 AM 02:44
이벤트: 보완요구서한 (CRL)
방향: 부정적
기업: RGNX리젠엑스바이오
자산: clemidsogene lanparvovec
출처: SEC 8-K
신뢰도: 출처 확인
캘린더: FDA 심사 결정 (PDUFA) · 2026년 2월 8일 · 보완요구서한 (CRL)

출처 발췌

EX-99.1 2 rgnx-ex99_1.htm EX-99.1 EX-99.1 EXHIBIT 99.1 REGENXBIO Announces Regulatory Update on RGX-121 BLA for MPS II • FDA issues Complete Response Letter for RGX-121 (clemidsogene lanparvovec) for treatment of Mucopolysaccharidosis II (MPS II) • REGENXBIO plans to work with FDA on a path forward with the goal of resubmitting the BLA ROCKVILLE, Md., February 9, 2026 – REGENXBIO Inc. (Nasdaq: RGNX) today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) regarding its Biologics License Application (BLA) for RGX-121 (clemidsogene lanparvovec) for the treatment of Mucopolysaccharidosis II (MPS II), an ultra-rare neurodegenerative disease also known as Hunter syndrome. In May 2025, the FDA accepted the RGX-121 BLA under the accelerated

SEC 8-K