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.
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.
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.
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.
