Precigen, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted full approval to PAPZIMEOS™ (zopapogene imadenovec-drba) for the treatment of adults with recurrent respiratory papillomatosis (RRP). This is a historic moment as PAPZIMEOS is the first and only FDA-approved therapy for RRP, a rare and debilitating disease caused by a chronic HPV 6 or HPV 11 infection.

PAPZIMEOS is a non-replicating, adenoviral vector-based immunotherapy designed to generate an immune response that targets the HPV 6/11 proteins, which are the root cause of RRP. The therapy is administered via four subcutaneous injections over a 12-week period. Unlike the previous standard of care, which involved repeated surgeries to remove tumors, PAPZIMEOS aims to provide a long-lasting therapeutic effect by addressing the underlying cause of the disease.

The full approval was based on data from a pivotal open-label, single-arm clinical study. The results showed that PAPZIMEOS was effective and well-tolerated. 51% of patients in the study achieved a complete response, meaning they required no surgeries for 12 months after treatment. This response was durable, with many patients remaining surgery-free for over two years. The treatment's safety profile was favorable, with common side effects being mild to moderate injection site reactions, fatigue, chills, and fever.

The approval of PAPZIMEOS marks a major advancement for the estimated 27,000 adult RRP patients in the U.S. Precigen is immediately beginning commercialization efforts and has launched a patient support program, Papzimeos SUPPORT, to assist with insurance navigation and financial assistance. The company will also hold a conference call on Monday, August 18, to provide more details about the approval and commercialization strategy.