The commercial-stage precision medicines company was just granted orphan drug exclusivity by the FDA for PAPZIMEOS™ (zopapogene imadenovec-drba), its treatment for RRP, a disease that typically requires patients to undergo multiple surgeries each year to remove benign tumors called papillomas that grow in the respiratory tract.

Orphan drug exclusivity is granted to certain drugs and biologics approved for rare diseases or conditions that affect fewer than 200,000 people in the United States. The orphan drug exclusivity means the FDA can't approve any similar drugs to treat RRP for seven years, extending PAPZIMEOS' market exclusivity through August 2032.

"This regulatory exclusivity, together with Precigen's patent portfolio covering PAPZIMEOS and its therapeutic use, enhances the product's value by strengthening market protection and long-term revenue potential, which in turn supports continued innovation for rare diseases," said Helen Sabzevari, PhD, President and CEO of Precigen.

How It Works

PAPZIMEOS uses a modified adenovirus to teach the immune system to spot and attack the proteins found in HPV 6 and HPV 11, which are the main cause of RRP. PAPZIMEOS reduces the number of surgeries a patient needs and provides a more effective way of clearing the airway, reports the company. Discovered, designed and developed in Precigen's labs using its proprietary AdenoVerse therapeutic platform, the company says PAPZIMEOS represents a new therapeutic paradigm for RRP.

The drug is commercially available in the U.S. and is being prescribed nationwide across both major medical centers and community practices, with patients spanning a range of disease severities actively receiving treatment.

Encouraged By Long-Term Study Results

The orphan drug exclusivity of PAPZIMEOS comes at a time when Precigen released encouraging long-term durability data from its pivotal study at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago.

The data showed that 15 out of 18 complete responders, or 83%, demonstrated ongoing complete responses for at least 36 months without any additional treatment for RRP. Five didn't need treatments after four years. Participants did not receive any additional treatments for RRP, including surgery or off-label investigational treatments, during the follow-up period, and no new safety events were observed during long-term follow-up.

"The presentation at ASCO marks an important maturation of the PAPZIMEOS pivotal study data, with all complete responders now followed for at least 36 months, 83% with ongoing response, including 5 patients who are surgery-free beyond 4 years," said Dr. Sabzevari. "For adults living with RRP, durability matters. These results continue to show that PAPZIMEOS provides sustained complete responses, reinforcing its role as the new standard of care for a disease historically managed through repeated surgeries." Follow-up for the study remains ongoing.

PAPZIMEOS gives RRP patients another option besides surgery, and now Precigen offers a solution with some protection from competition for the next several years. With promising long-term durability results added to the mix, Dr. Sabzevari shared updates on the launch when she spoke at last week's Goldman Sachs 47th Annual Global Healthcare Conference.

Contact:
Steven Harasym
investors@precigen.com

Click here for more information on Precigen and PAPZIMEOS

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