Zopapogene Imadenovec-Drba for Recurrent Respiratory Papillomatosis

To summarize the clinical evidence for zopapogene imadenovec-drba (Papzimeos, Precigen Inc) in the treatment of recurrent respiratory papillomatosis (RRP), address pharmacy-specific operational considerations for this drug and provide an overview of emerging human papillomavirus (HPV)-directed immunotherapies that may expand the pharmacist's role in RRP management.

PubMed, Scopus, Google Scholar, and ClinicalTrials.gov (through May 2026); prescribing information; treatment guidelines; AMCP Format 5.0 dossier; and FDA clinical review.

Efficacy and safety data were derived from the pivotal PRGN-2012-201 trial (NCT04724980). Pipeline therapies were identified from IPD Analytics.

In the phase 1/2 PRGN-2012-201 trial (n = 35 at recommended dose), zopapogene imadenovec demonstrated a 51% complete response rate at 12 months (43% at 24 months), an objective response rate of 66%, and no serious treatment-related adverse events. INO-3107, a DNA immunotherapy under FDA review, has shown complete response rates from 28% to 50% by year 2.Relevance to Patient Care and Clinical Practice in Comparison to Existing Drugs:Zopapogene imadenovec-drba is the first FDA-approved therapy for RRP, a condition historically managed through repeated surgical debulking. Off-label adjuvant therapies such as intralesional cidofovir, bevacizumab, and HPV vaccination have been used with variable efficacy.

The shift from primarily surgical management of RRP to HPV-directed immunotherapy creates an expanded role for pharmacists in high-cost drug stewardship (including formulary evaluation, manufacturer and payer engagement, and revenue cycle optimization), operational management (including procurement, ultra-cold storage, and biosafety handling), clinical monitoring, and patient education on a mechanistically novel therapy.