REGENXBIO Announces Alignment with FDA on Path Forward for NAVSUNLI™ BLA Resubmission for Accelerated Approval; First Potential Gene Therapy for MPS II
REGENXBIO secured a critical regulatory win, aligning with the FDA on an accelerated approval path for NAVSUNLI without requiring additional clinical studies or a control arm. The company plans to request a Type A meeting in July and resubmit the BLA in Q3 2026, with the FDA committing to an expedited review of the existing data.
Key figures
- Drug Name
- NAVSUNLI (clemidsogene lanparvovec-sngl, RGX-121)
- Annual incidence
- more than 500
- Patients worldwide
- Approximately 2,000
- Bla resubmission timeline
- Q3 2026
AI analysis
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