US FDA Approves PAPZIMEOS, Expands Treatment Options

US FDA Approves PAPZIMEOS: The US Food and Drug Administration (FDA) has approved PAPZIMEOS, a treatment for patients with schizophrenia. The approval is based on clinical trials that showed the medication was effective and well-tolerated. This is a significant development for the pharmaceutical industry and a positive step forward for patients with schizophrenia.

The U.S. Food and Drug Administration (FDA) has granted full approval to Precigen’s PAPZIMEOS (zopapogene imadenovec-drba) for the treatment of adults with Recurrent Respiratory Papillomatosis (RRP). This marks a significant milestone in the treatment landscape for RRP, a rare and life-threatening condition that affects approximately 27,000 adults in the U.S.

PAPZIMEOS is a non-replicating adenoviral vector-based immunotherapy that targets the root cause of RRP, which is caused by human papillomavirus (HPV) types 6 and 11. The therapy demonstrated remarkable efficacy in clinical trials, with 51% of patients achieving a complete response, requiring no surgeries for 12 months post-treatment. Of these, 15 patients maintained a complete response at 24 months [1].

The pivotal clinical trial, conducted by the National Institutes of Health (NIH), enrolled 38 patients and evaluated the safety and tolerability of PAPZIMEOS. The trial showed no dose-limiting toxicities and no treatment-related adverse events higher than grade 2. The most common adverse events included injection site reactions, fatigue, chills, and fever [2].

The FDA’s full approval of PAPZIMEOS signifies a major regulatory win for Precigen. Unlike accelerated approval, which requires a confirmatory clinical trial, this approval was granted based on compelling efficacy data. This decision reduces Precigen’s post-approval obligations and accelerates the therapy’s commercial timeline [3].

The approval comes with a broad label covering all adult RRP patients, maximizing the addressable market. PAPZIMEOS’ unique mechanism as a non-replicating adenoviral vector-based immunotherapy targeting HPV 6/11 gives it a first-in-class designation with no competitive threats on the horizon [2].

Precigen has established a comprehensive patient support program, Papzimeos SUPPORT, to facilitate market access and demonstrate commercial readiness. This approval validates Precigen’s AdenoVerse platform technology, potentially supporting additional indications and enhancing the company’s pipeline value beyond this single product [3].

References:
[1] 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. PR Newswire. August 15, 2025. Accessed August 15, 2025. https://prnmedia.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
[2] Adjuvant PRGN-2012 in Adult Patients With Recurrent Respiratory Papillomatosis. Clinicaltrials.gov. Accessed August 15, 2025. https://clinicaltrials.gov/study/NCT04724980
[3] Precigen's PAPZIMEOS becomes first FDA-approved therapy for RRP, offering durable 51% complete response rates and eliminating costly surgeries. AINVEST. August 15, 2025. Accessed August 15, 2025. https://www.ainvest.com/news/precigen-fda-approval-papzimeos-game-changer-rrp-market-catalyst-shareholder-2508/