PLYMOUTH MEETING, PA., October 31, 2026 — The U.S. Food and Drug Administration has granted accelerated approval to VOCERYQ, a plasmid DNA-based immunotherapy designed to reduce the frequency of surgery in adults with recurrent respiratory papillomatosis. The decision marks the first FDA approval of a DNA medicine in the United States.
The Burden of RRP and the Cycle of Surgery
Recurrent respiratory papillomatosis is a rare disease caused by human papillomavirus types 6 and 11. Benign tumors form along the aerodigestive tract, narrowing the airway and causing hoarseness, shortness of breath, and stridor. According to medical literature, the condition affects approximately 1.8 adults per 100,000 annually. For decades, the standard of care has relied on repeated surgical excisions to remove papillomas and maintain airway patency. Every procedure carries inherent risks of laryngeal injury, scarring, and voice changes. Patients often face an exhausting cycle of operations, managing the disease but never truly escaping it.
In the RRP-001 trial, the 26 patients who made up the efficacy population had a median of 4.5 surgeries in the 12 months before enrollment, with a range of three to eight procedures. That baseline illustrates the relentless surgical burden that defines life with RRP. Simon Best, Associate Professor of Otolaryngology at Johns Hopkins University and a lead investigator for the study, said the approval represents an important step in improving quality of life because every surgery carries the risk of laryngeal injury. He added that he believes it is in the best interest of patients that the standard of care transition away from surgical interventions to immunotherapy after diagnosis.
How VOCERYQ Functions as a DNA-Based Immunotherapy
VOCERYQ, known generically as doruxapapogene ralaplasmid and lalikinogene sifuplasmid-gxfd, is the first plasmid DNA-based immunotherapy approved in the U.S. The therapy delivers two plasmids via intramuscular injection. One plasmid encodes antigens for HPV-6 and HPV-11, targeting the viral root cause of the disease. The second plasmid encodes the immune-enhancing cytokine interleukin-12, or IL-12, designed to stimulate a targeted immune response against the infected cells.
The injection is administered using the CELLECTRA 5PSP device, which employs electroporation — brief electrical pulses — to help the DNA enter cells. The FDA approved the drug and the device together. INOVIO, the Plymouth Meeting-based biotechnology company behind the treatment, describes the platform as a novel DNA medicine technology validated by this approval. Dr. Jacqueline Shea, INOVIO’s President and Chief Executive Officer, said the therapy is designed to deliver what matters most to patients: a reduction in the surgical burden caused by RRP.
Clinical Trial Results and Reduction of Surgical Frequency
The accelerated approval is based on data from the open-label, multicenter RRP-001 study. The trial enrolled 32 adults who had undergone at least two papilloma excision surgeries in the prior 12 months, including a surgery scheduled at trial entry. Before the first dose, patients received standard-of-care surgical debulking of visible papillomas. A nine-week dosing regimen followed, during which the protocol required no additional excision surgeries.
The primary efficacy population consisted of 26 patients who had three or more surgeries in the year before enrollment. In this group, seven patients — 27 percent, with a 95 percent confidence interval of 12 to 48 percent — achieved a complete response. Complete response was defined as no requirement for surgical intervention through 12 months of follow-up. The trial had no control arm. The FDA based its decision on the reduction in surgery frequency, an intermediate clinical endpoint the agency considers reasonably likely to predict clinical benefit. According to INOVIO, complete responses of this kind are not expected in the untreated natural history of the disease.
The Shift Toward Immunotherapy as a New Standard of Care
The approval comes a little more than a year after the FDA approved zopapogene imadenovec-drba, marketed as Papzimeos, an adenoviral vector-based immunotherapy for adults with RRP. VOCERYQ offers a distinct mechanism using plasmid DNA rather than a viral vector. For families navigating this disease, the arrival of a second immunotherapy option signals a genuine shift in the treatment paradigm. The goal is no longer just managing airway obstruction through the operating room but engaging the immune system to control the viral drivers of the disease.
INOVIO has stated its belief that VOCERYQ has the potential to become a new standard of care for this devastating disease. The company acknowledged the patients, study investigators, and the RRP Foundation for their roles in reaching this milestone. Continued approval for this indication may be contingent on verification and description of clinical benefit in confirmatory studies, a standard requirement for drugs approved under the accelerated pathway.
Availability and the Path to Commercial Launch
INOVIO expects to make VOCERYQ available to order and ship in approximately 60 days from the October 30 announcement. A full commercial launch is planned for the first quarter of 2027. The company hosted a conference call on November 2, 2026, to discuss the approval and commercial plans. As this new option reaches clinics, patients and caregivers should discuss the potential benefits and risks with their treating physicians to determine if VOCERYQ is appropriate for their specific situation.
The Patient Experience and Quality of Life Impact
Recurrent respiratory papillomatosis imposes a significant physical and emotional toll on those affected, extending far beyond the operating room. Patients frequently report a diminished quality of life due to chronic symptoms such as hoarseness, dysphagia, and stridor, which can interfere with basic functions like speaking and eating. The psychological burden is equally profound, as individuals navigate the anxiety of unpredictable disease flares and the frustration of a treatment paradigm dominated by repeated interventions. Medical literature indicates that the condition affects approximately 1.8 adults per 100,000 annually, classifying it as a rare disease, yet its impact on daily living is substantial. For many, the “exhausting cycle of repeated surgeries” described by study investigators becomes a defining aspect of their identity and routine, necessitating frequent time off work and school for procedures that offer only temporary relief.
The transition toward immunotherapy, such as VOCERYQ, represents a hopeful shift in managing this chronic burden. By targeting the HPV-6 and HPV-11 viral antigens that drive tumor growth, the therapy aims to reduce the body’s reliance on the scalpel. Simon Best, Associate Professor of Otolaryngology at Johns Hopkins University and a lead investigator for the RRP-001 trial, emphasized that the approval is an important step in improving quality of life because every surgery carries the risk of laryngeal injury. He further stated that transitioning the standard of care away from surgical interventions toward immunotherapy after diagnosis is in the best interest of patients. This potential to break the cycle of surgery offers not only a reduction in physical risks but also the psychological benefit of a more sustainable long-term management strategy for a disease that has historically been defined by its recurrence.
Sources
- INOVIO Announces FDA Accelerated Approval of VOCERYQ™, an Immunotherapy to Reduce the Frequency of Surgery in Adults with Recurrent Respiratory Papillomatosis (RRP) – AOL
- Laryngeal papillomatosis
- Voceryq Gains FDA Accelerated Approval for Recurrent Respiratory Papillomatosis | HCPLive
Photo: David P Howard (CC BY-SA 2.0)
