FDA Approval of Papzimeos Marks Historic Breakthrough for Adults with Recurrent Respiratory Papillomatosis
The Recurrent Respiratory Papillomatosis Foundation (RRPF) has announced a landmark achievement in the fight against Recurrent Respiratory Papillomatosis (RRP): the U.S. Food and Drug Administration (FDA) has granted full approval to Papzimeos (zopapogene imadenovec-drba), a novel immunotherapy developed by Precigen. The approval, which was conducted under Priority Review, designates Papzimeos as the first and only FDA-approved treatment for RRP in adults.
This decision represents more than a scientific milestone—it symbolizes decades of resilience, advocacy, and dedication from the global RRP community. For thousands of patients who have endured years of invasive surgeries and uncertain futures, Papzimeos introduces a new era of treatment, one rooted in precision science and immune system empowerment rather than repeated surgical intervention.
Understanding Recurrent Respiratory Papillomatosis (RRP)
Recurrent Respiratory Papillomatosis is a rare, chronic disease caused by human papillomavirus (HPV) types 6 and 11. It is characterized by the growth of benign tumors, or papillomas, along the respiratory tract, particularly in the larynx and vocal cords. These growths can obstruct the airway, leading to symptoms such as hoarseness, chronic coughing, breathing difficulties, and in severe cases, life-threatening airway obstruction.
Traditionally, RRP has been managed almost exclusively through surgery, where papillomas are removed to restore breathing and preserve vocal function. However, papillomas almost invariably return, forcing patients—sometimes even children—to undergo dozens or even hundreds of surgeries over their lifetime.
While surgical techniques have improved, and adjuvant therapies have been explored, there has never before been an FDA-approved medical therapy targeting the disease at its root cause. This made RRP one of the most burdensome and emotionally exhausting conditions for patients and families.
The Significance of Papzimeos Approval
Papzimeos is a non-replicating adenoviral vector-based immunotherapy engineered to generate an immune response against papilloma cells that express HPV types 6 and 11, the same viral strains responsible for nearly all cases of RRP.
- First FDA-approved treatment for RRP: Until now, patients had no non-surgical therapeutic option.
- Approved for adults: The FDA decision currently applies to adult patients with RRP.
- Targets the underlying cause: Unlike surgery, which removes papillomas temporarily, Papzimeos works at the biological level to stimulate an immune response designed to eliminate the root viral driver of papilloma formation.
- Dosing regimen: Treatment is administered as four subcutaneous injections over a 12-week interval, offering a structured, short-term protocol compared with the lifelong cycle of surgical interventions.
This approval provides hope not just for improved outcomes, but also for reducing the emotional, physical, and financial toll associated with repeated surgical procedures.
A Community’s Hope Realized
The approval of Papzimeos is more than just a regulatory success—it is a moment of healing and validation for patients, caregivers, clinicians, and advocates who have spent decades pushing for scientific progress in RRP.
“For decades, our community has persevered through an isolating journey of countless surgeries, silence, and uncertainty,” said Kim McClellan, President of the Recurrent Respiratory Papillomatosis Foundation (RRPF). “Yet while we continued to breathe, we continued to hope. Today, our hope is realized. With the approval of Papzimeos, our hope is no longer a dream—it’s a reality. This is a moment of profound healing, not just for the body, but for the spirit of every adult living with RRP. We finally have an FDA-approved treatment that targets the root cause of RRP, giving many members of our community the chance to reclaim lives unencumbered by constant surgeries.”
McClellan’s words echo the sentiment of countless families who have endured the uncertainty and trauma of living with RRP. Every surgery carried risks. Every return of papillomas meant renewed anxiety. With Papzimeos, there is now tangible proof that science can replace fear with possibility.
Decades of Advocacy and Research
The FDA’s decision was the culmination of decades of tireless work by patients, researchers, clinicians, and advocacy organizations. RRPF and its partners have long championed awareness of RRP, supported clinical research, and amplified the voices of patients whose journeys often remained in the shadows of public health discussions.
The development of Papzimeos also reflects the collaborative power of science and community. Investigators, clinicians, and National Institutes of Health (NIH) collaborators played key roles in advancing the scientific understanding of RRP and testing innovative therapeutic strategies. Industry partners, particularly Precigen, invested in research and clinical development, helping transform years of advocacy into a tangible, life-changing therapy.
A Deeply Personal Victory
While regulatory approvals are often discussed in terms of scientific merit or business implications, the FDA’s approval of Papzimeos is profoundly personal for those living with RRP. Each milestone achieved represents more than data points on a clinical trial—it represents voices preserved, breaths restored, and lives regained.
The RRPF emphasized that the approval is a victory for every surgery endured, every voice that spoke up, and every patient who held on to hope despite the relentless nature of the disease. It is not just a new treatment—it is a recognition of the suffering endured and a commitment to a brighter future.
Beyond the U.S.: The Next Frontier
While the FDA approval is a historic step, the RRPF underscored that this is not the final destination. The foundation remains committed to ensuring that all patients, including children and those outside the United States, can access safe and effective treatments.
- Pediatric patients: Currently, there is no FDA-approved treatment for children with RRP. Pediatric cases often present earlier and can be more aggressive, requiring frequent surgical interventions. The approval of Papzimeos for adults sets a precedent, but the urgent need for pediatric options remains.
- Global access: RRP is not confined to the U.S. Families around the world face the same burdens. Advocacy must now focus on expanding clinical trials, regulatory submissions, and collaborations to bring Papzimeos—or similar therapies—to international patients.
This broader vision reflects RRPF’s mission to advance research, advocate for equitable access, and push science forward until every patient, regardless of age or geography, has a pathway to healing.
Renewed Commitment from RRPF
The RRPF stated that the approval strengthens their resolve to continue their work with even greater urgency. The foundation will:
- Support ongoing research into RRP and related conditions.
- Advocate for pediatric trials to extend treatment options to children.
- Work with global partners to ensure broader access.
- Amplify patient voices, ensuring their lived experiences remain at the center of scientific and policy discussions.
By focusing on these priorities, the RRPF aims to transform this milestone into a foundation for broader, global impact.
A Turning Point in RRP Care
The approval of Papzimeos is more than a new therapy—it represents a turning point in how RRP is understood and managed. For the first time, patients have an option beyond surgery, a treatment that strikes at the biological roots of the disease rather than just addressing its physical manifestations.
For patients, families, and clinicians, it is the beginning of a new chapter—one where hope is no longer deferred, but realized.
