Shots:
- The US FDA has accepted BLA & granted priority review to RGX-121 (clemidsogene lanparvovec) for the treatment of Mucopolysaccharidosis II (MPS II or Hunter syndrome), with PDUFA action date of Nov 09, 2025
- As per Jan 2025 partnership, NS Pharma (Nippon Shinyaku’s subsidiary) will commercialize RGX-121 in the US post approval while REGENXBIO will handle commercial manufacturing & supply chain. Also, REGENXBIO will retain all rights to & proceeds from any potential PRV granted upon approval
- RGX-121, a one-time AAV therapy for boys with MPS II, is designed to deliver the IDS gene to the CNS, allowing long-term I2S protein production beyond the blood-brain barrier for sustained cross-correction of CNS cells
Ref: PRNewswire | Image: REGENXBIO | Press Release
Related News:- REGENXBIO Join Forces with Nippon Shinyaku to Develop and Commercialize RGX-121 and RGX-111 for Mucopolysaccharidosis (MPS)
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