FDA Clears Path for Regenxbio’s Hunter Syndrome Gene Therapy, BLA Resubmission Q3 2026
RGNX•The FDA has agreed to reconsider Regenxbio’s Navsunli gene therapy for Hunter syndrome using the existing clinical package, eliminating the need for additional patient enrollment or new studies. Regenxbio plans a July meeting with the FDA and aims to resubmit its Biologics License Application in Q3 2026.
1. FDA Reassessment of Navsunli
The FDA has reversed its February 2026 complete response letter on Navsunli (clemidsogene lanparvovec-sngl), agreeing the existing clinical dataset is sufficient for accelerated approval review and waiving requirements for additional patient enrollment or new studies.
2. Path to BLA Resubmission
Regenxbio completed a collaborative discussion with the FDA, scheduling a July meeting to review long-term biomarker and clinical data before targeting a Biologics License Application resubmission in Q3 2026.
3. Broader Regulatory Shift
This decision follows a similar reversal for uniQure’s Huntington’s therapy and coincides with recent leadership changes at CBER and the FDA commissioner position, highlighting a more flexible regulatory stance on rare disease submissions.
