REGENXBIO To Resubmit NAVSUNLI BLA in Q3 After FDA Waives New Studies
RGNX•REGENXBIO secured FDA agreement to resubmit NAVSUNLI’s BLA in Q3 2026 with no new patient enrollment or studies required. FDA plans to reverse its earlier rejection of the gene therapy, initiate expedited review under the accelerated approval pathway and hold a Type A meeting in July to assess CAMPSIITE longer-term data.
1. FDA Waives Additional Study Requirements
FDA confirmed REGENXBIO need not enroll additional patients or conduct new studies, including an untreated control arm, and will consider existing long-term biomarker and clinical data from the CAMPSIITE study for accelerated approval of NAVSUNLI.
2. Q3 2026 BLA Resubmission and July Type A Meeting
REGENXBIO expects to hold a Type A meeting with FDA in July 2026 and to resubmit the NAVSUNLI BLA rapidly thereafter in Q3 2026, initiating expedited review and commencing labeling discussions.
3. Potential Accelerated Approval and Priority Review Voucher
FDA’s expedited review under the accelerated approval pathway could lead to the first one-time gene therapy for Hunter syndrome, with potential receipt of a Priority Review Voucher upon approval, of which REGENXBIO retains all proceeds.
4. Commercial Partnership and Pipeline Implications
Under the January 2025 strategic partnership, NS Pharma will commercialize RGX-121 in the U.S. post-approval, strengthening REGENXBIO’s late-stage pipeline alongside RGX-202 and ABBV-RGX-314 and highlighting its AAV platform leadership.
