REGENXBIO: FDA naglagay ng clinical hold sa RGX-121 BLA

Naglagay ang FDA ng clinical hold sa RGX-121 BLA matapos makita ang asymptomatic spine MRI findings sa limang kalahok ng CAMPSIITE. Hindi inaasahan ng kumpanya na muling isumite ang BLA sa malapit na panahon at isasama nila ang feedback ng FDA at mas mahabang-term data bago ang susunod na hakbang.

Mahahalagang fact

Na-file: Ago 24, 2026, 09:20 PM
Kaganapan: Inspeksyon sa manufacturing
Direksyon: Negatibo
Kumpanya: RGNXREGENXBIO
Asset: clemidsogene lanparvovec
Pinagmulan: SEC 8-K
Kumpiyansa: May source

Sipi mula sa source

EX-99.1 2 rgnx-ex99_1.htm EX-99.1 EX-99.1 EXHIBIT 99.1 REGENXBIO Announces Regulatory Update on RGX-121 for MPS II ROCKVILLE, Md., August 24, 2026 – REGENXBIO Inc. (Nasdaq: RGNX) today provided an update on its investigational gene therapy, RGX-121 (clemidsogene lanparvovec), for the treatment of Mucopolysaccharidosis type II (MPS II), also known as Hunter Syndrome. The U.S. Food and Drug Administration (FDA) placed a clinical hold on RGX-121 following the discovery of asymptomatic spine MRI findings in five participants in the CAMPSIITE ® study; REGENXBIO does not expect to resubmit the RGX-121 Biologics License Application (BLA) in the near term. “We believe these findings are unique and limited to our Hunter Syndrome program, and require longer-term follow-up and additional data an

SEC 8-K