Precigen, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted full approval to PAPZIMEOS (zopapogene imadenovec-drba), the first and only FDA-approved treatment for adults with recurrent respiratory papillomatosis (RRP). This marks a significant milestone for patients suffering from the rare and debilitating disease caused by chronic infection with human papillomavirus (HPV) types 6 and 11.
RRP leads to severe respiratory issues, including voice problems, airway obstruction, and recurrent infections. Until now, management relied largely on repeated surgeries, which do not target the underlying cause and carry substantial risks and burdens for patients and healthcare systems.
PAPZIMEOS is a novel adenoviral vector-based immunotherapy designed to express a fusion antigen targeting HPV 6 and 11 proteins. The treatment consists of four subcutaneous injections over 12 weeks, aiming to address the root cause of RRP.
Helen Sabzevari, President and CEO of Precigen, highlighted the historic nature of the approval: “For more than a century, patients have relied on repeated surgeries. Today, PAPZIMEOS offers the first therapy to target the disease’s root cause.” She also credited the AdenoVerse platform and collaboration with the NIH and FDA for accelerating development.
The approval was based on data from a pivotal open-label study involving 35 adult RRP patients, where 51% achieved a durable complete response, requiring no surgeries for over a year. PAPZIMEOS was well-tolerated with no serious treatment-related adverse events.
Kim McClellan, President of the Recurrent Respiratory Papillomatosis Foundation, called the approval “a momentous milestone,” offering hope to patients and families facing a relentless disease.
Precigen plans immediate promotion of PAPZIMEOS and has launched a patient support program to assist with insurance, financial aid, and ongoing access.
This approval ushers in a new era of treatment for RRP, focusing on addressing the disease at its source rather than merely managing symptoms.