REGENXBIO announced FDA's extension of the BLA review timeline for RGX-121, a potential one-time therapy for Hunter syndrome.
Commercial launch plans for RGX-121 remain on track, with pivotal data set to be presented in September 2025.
The extension was due to the submission of positive 12-month clinical data for all patients in the pivotal study of RGX-121, addressing neurodevelopmental decline in Hunter syndrome patients.
FDA Review Extension
The FDA extended the PDUFA goal date for RGX-121 from November 9, 2025, to February 8, 2026, following the submission of longer-term clinical data.
Commercial Launch Plans
REGENXBIO's plans for the commercial launch of RGX-121 remain unaffected by the timeline extension, ensuring timely access for patients.
Clinical Data Presentation
Updated pivotal data for RGX-121, including 12-month clinical insights, will be shared at the ICIEM meeting in September 2025.
- The approval delay allows REGENXBIO to provide comprehensive long-term data to support the efficacy and safety of RGX-121.
- RGX-121's unique approach of addressing the genetic cause of Hunter syndrome could potentially revolutionize the treatment landscape for MPS II patients.
With the FDA's extended review timeline and forthcoming data presentation, REGENXBIO's commitment to advancing gene therapies for rare diseases like Hunter syndrome remains unwavering.