Precigen Receives FDA Approval for Papzimeos, First Treatment for Recurrent Respiratory Papillomatosis
核心洞察
Precigen's Papzimeos (搜索) (zopapogene imadenovec-drba (搜索)) becomes the first and only FDA-approved treatment for adults with recurrent respiratory papillomatosis (搜索) (RRP (搜索)), a rare HPV-related airway condition.
The gene therapy demonstrated a 51% complete response rate in a pivotal phase I/II study, with 18 of 35 patients eliminating the need for surgeries in the 12 months following treatment.
The FDA granted full approval rather than accelerated approval, meaning Precigen will not need to conduct confirmatory studies for the RRP (搜索) indication.
Precigen Inc. has achieved a significant regulatory milestone with the FDA's approval of Papzimeos (搜索) (zopapogene imadenovec-drba (搜索)), marking the first and only approved treatment for adults with recurrent respiratory papillomatosis (搜索) (RRP (搜索)). The approval has driven Precigen's stock to surge 144.4% in August, with shares jumping over 50% in premarket trading following the announcement.
Breakthrough Gene Therapy for Rare Disease
Papzimeos (搜索) represents a landmark achievement for the RRP (搜索) patient community, addressing a rare condition that affects approximately 27,000 adult patients in the United States and more than 125,000 patients globally. RRP is a rare, non-cancerous viral tumor condition in the airways caused by human papillomavirus (HPV) types 6 and 11, which can be potentially life-threatening.
The gene therapy, developed using Precigen's proprietary AdenoVerse platform, is a non-replicating adenoviral vector immunotherapy engineered to address the primary drivers of RRP (搜索). The treatment is administered through four subcutaneous injections given over 12 weeks and has received both breakthrough therapy and orphan drug designations from the FDA.
Strong Clinical Trial Results
The FDA approval was based on data from a pivotal phase I/II study that met both primary safety and pre-specified primary efficacy endpoints. In the study, 51% of patients (18 of 35) achieved a complete response, eliminating the need for surgeries in the 12 months following Papzimeos (搜索) treatment.
The responses demonstrated remarkable durability, with 15 of the 18 patients assessed at 24 months maintaining a complete response beyond one year. The drug was well-tolerated in the RRP (搜索) patient population, with only mild to moderate treatment-related adverse events reported.
Full Approval Advantage
Notably, the FDA granted full approval rather than accelerated approval, which means Precigen will not be required to conduct confirmatory studies for the RRP (搜索) indication. The company completed submission of the rolling biologics license application (BLA) for Papzimeos (搜索) in December 2024 under the FDA's accelerated approval pathway, but the agency ultimately provided full approval based on the strength of the clinical data.
Addressing Significant Unmet Medical Need
Currently, there is no known cure for RRP (搜索), and typical treatment involves frequent surgeries that do not target the root cause of the disease and often lead to considerable complications. RRP causes benign tumors called papillomas to grow in the respiratory tract, most commonly in the larynx (voice box), caused by HPV types 6 and 11.
Commercial Launch and Market Competition
Precigen is set to initiate the commercial launch of Papzimeos (搜索) immediately, with the company rolling out Papzimeos SUPPORT, a patient services platform designed to streamline insurance processes, reduce financial barriers, and ensure sustained access to treatment.
The company faces potential competition from Inovio Pharmaceuticals, which is developing INO-3107, an investigational DNA medicine designed to generate an antigen-specific T-cell response against both HPV-6 (搜索) and HPV-11 (搜索) proteins. Inovio is on track to begin a rolling BLA submission for INO-3107 soon and plans to seek accelerated approval for a potential 2026 launch, pending FDA approval. Like Papzimeos (搜索), INO-3107 has also received breakthrough therapy and orphan drug designations from the FDA.