Precigen snags priority review, August PDUFA date for gene therapy

The U.S. FDA has accepted and granted priority review to Precigen’s BLA for its adenovirus-based gene therapy, PRGN-2012, for the treatment of recurrent respiratory papillomatosis (RRP), a rare disease characterized by the recurrent growth of benign tumors in the respiratory tract.

If approved, PRGN-2012 would be the first and only FDA-approved therapeutic for the treatment of adults with RRP. RRP is a difficult-to-treat, lifelong neoplastic disease of the upper and lower respiratory tracts caused by infection with human papillomavirus (HPV) type 6 or HPV type 11 that can be fatal. Currently, there is no cure for RRP and the current standard-of-care is repeated surgeries, which do not address the underlying cause of disease and are associated with significant morbidity.

PRGN-2012 is a gene therapy with optimized antigen design that uses Precigen’s gorilla adenovector technology, part of the company’s proprietary AdenoVerse platform, to elicit immune responses directed against cells infected with HPV 6 or HPV 11.

Precigen's BLA is supported by data from the pivotal phase 1/2 clinical study which were presented at the 2024 ASCO annual meeting and published in The Lancet Respiratory Medicine. The study met its primary safety and efficacy endpoints, with more than 50% of patients achieving complete response and more than 85% of patients experiencing a decrease in surgical interventions in the year after PRGN-2012 treatment compared to the year prior to treatment.

The FDA set a PDUFA target action date of August 27, 2025 for PRGN-2012 and has indicated that the agency is not currently planning to hold an advisory committee meeting to discuss the application.