FDA approves first immunotherapy for recurrent respiratory papillomatosis
Drug Approval

FDA approves first immunotherapy for recurrent respiratory papillomatosis

  • By IPP Bureau | August 19, 2025

The U.S. Food and Drug Administration (FDA) has approved Papzimeos (zopapogene imadenovec-drba), the first non-replicating adenoviral vector-based immunotherapy for the treatment of adult patients with recurrent respiratory papillomatosis (RRP).

RRP is a rare, chronic disease caused by persistent infection with human papillomavirus (HPV) types 6 or 11, leading to benign tumor growth in the respiratory tract, most often in the larynx. The condition can cause serious morbidity, including voice changes, breathing difficulties, and airway obstruction. Until now, there have been no approved medical therapies to eliminate the need for repeated surgical procedures.

“Randomized trials are not always needed to approve medical products and this approval is proof of that philosophy,” said Vinay Prasad, Director of the FDA’s Center for Biologics Evaluation and Research (CBER).

“The FDA will always demand the correct clinical study for the specific medical product and disease. Our requirements for products given to tens of millions of healthy people will be different than products given to at most hundreds or thousands of patients with unique diseases.”

RRP affects an estimated 1,000 new patients each year in the U.S., highlighting the significant unmet need. Papzimeos is administered by subcutaneous injection and is designed to stimulate an immune response against HPV 6/11-infected cells, offering a novel mechanism distinct from surgical interventions.

“This approval has the potential to transform the treatment landscape for RRP and offer lasting relief for patients who previously faced repeated surgeries to control symptoms of their disease,” said Vijay Kumar, Acting Director of the Office of Therapeutic Products in CBER.

The FDA’s decision is based on results from a single-arm, open-label study of adult patients with RRP requiring three or more surgeries per year. Patients received four subcutaneous injections of Papzimeos over 12 weeks after surgical debulking procedures.

Papzimeos demonstrated a favorable safety profile, with most treatment-emergent adverse events reported as mild to moderate. No dose-limiting toxicities or treatment-related serious adverse events were observed.

The approval was granted under Priority Review, with Orphan Drug and Breakthrough Therapy designations. The FDA granted approval of Papzimeos to Precigen, Inc.

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