FDA Grants Full Approval to Precigen’s Papzimeos for Rare Disease RRP
On August 15, 2025, the FDA granted full approval to Precigen's Papzimeos (zopapogene imadenovec-drba), the first and only approved therapy for adults with recurrent respiratory papillomatosis (RRP)1345.
RRP is a rare disease caused by HPV that leads to benign tumors in the respiratory tract; up to 27,000 adults in the US may be affected13.
Papzimeos approval was based on results from a single-arm, open-label pivotal study:
51% of participants achieved complete response (no need for surgery for 12 months), with durability up to 24 months34.
Papzimeos was well-tolerated:
most side effects were mild to moderate (pain, swelling at injection site, fatigue, chills, fever, muscle aches, headache, etc.), with no severe treatment-related adverse events or dose-limiting toxicities34.
FDA designated Papzimeos as an Orphan Drug and granted it Breakthrough Therapy status; the approval was processed under Priority Review4.
This therapy offers the potential to eliminate the need for repeated surgeries in RRP patients, addressing a significant unmet medical need and providing hope for those who previously had no effective treatment options1345.
Sources:
1. https://www.biopharminternational.com/view/fda-approves-precigen-immunotherapy-treat-recurrent-respiratory-papillomatosis
3. https://www.prnewswire.com/news-releases/precigen-announces-full-fda-approval-of-papzimeos-zopapogene-imadenovec-drba-the-first-and-only-approved-therapy-for-the-treatment-of-adults-with-recurrent-respiratory-papillomatosis-302530957.html
4. https://www.fda.gov/news-events/press-announcements/fda-approves-first-immunotherapy-recurrent-respiratory-papillomatosis
5. https://www.fiercepharma.com/pharma/precigen-scores-fda-nod-first-ever-treatment-hpv-related-disorder