The FDA approved Voceryq after 27% of trial patients with recurrent respiratory papillomatosis who had at least three prior surgeries skipped surgery for a year

Voceryq, a DNA-based immunotherapy from Inovio Pharmaceuticals, received accelerated approval from the Food and Drug Administration on Oct. 30 for adults with recurrent respiratory papillomatosis. In the study behind the decision, 7 of 26 patients, or 27%, needed no surgical intervention in the year after treatment. All 26 had undergone at least three surgeries in the 12 months before they enrolled.

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Recurrent respiratory papillomatosis, usually shortened to RRP, is a rare airway disease in which wart-like growths return after they are cut away. Patients can need operations every few weeks to keep breathing, or only once a year, and the disease has no cure.

A growth that returns after every operation

The National Institute on Deafness and Other Communication Disorders describes RRP as benign tumors, called papillomas, that grow in the airways from the nose and mouth down into the lungs. They form most often in the larynx and can grow quickly. The cause is infection with human papillomavirus types 6 and 11, and most people exposed to those viruses never develop the disease. Surgery with a laser or a microdebrider is the main treatment, and in the most severe cases a tracheotomy keeps the airway open.

Because the virus persists in tissue, hoarseness, chronic cough and voice changes come back along with the growths. Inovio estimates, from claims data, that at least 25,000 people in the U.S. are living with the condition. The RRP Foundation puts active cases nearer 20,000, and the NIDCD adds that adult-onset disease affects roughly two to three of every 100,000 adults, while the juvenile form is rarer. Each procedure carries its own risks, which is why a drug that spaces operations further apart has an obvious pull for patients who have had four or five in a year.

Two plasmids, an electrical pulse and a nine-week course

According to the FDA’s announcement, Voceryq is a recombinant plasmid DNA immunotherapy, the first of its kind cleared in the U.S. One plasmid carries instructions for HPV proteins; the second carries instructions for IL-12, a molecule that strengthens immune responses. The product targets HPV types 6 and 11 and is injected into muscle with the CELLECTRA 5PSP device, which uses brief electrical pulses to open cell membranes temporarily so the plasmids can enter.

Karim Mikhail, director of the FDA’s Center for Biologics Evaluation and Research, called the approval “a meaningful scientific breakthrough” and said it could mean fewer surgeries and fewer disruptions for patients.

In its own release, Inovio laid out the regimen: surgeons first remove visible papillomas by standard debulking, then the patient receives nine weeks of dosing with no further surgery required. Chief executive Jacqueline Shea said the goal is reducing the surgical burden RRP imposes. Simon Best, the Johns Hopkins investigator who led the trial, said every surgery carries a risk of laryngeal injury, which is the argument for treating the underlying infection instead of only cutting growths away. The median number of prior excisions among the 26 efficacy patients was 4.5, with a range of three to eight, so the people who reached the one-year mark without an operation had been among the most heavily treated in the study.

What accelerated approval does and does not settle

The agency granted the clearance under its accelerated approval pathway, which allows earlier approval for serious conditions on an intermediate endpoint that is reasonably likely to predict benefit. The manufacturer must then run confirmatory studies, and the FDA can move to withdraw a product if they fail. Voceryq also carries Breakthrough Therapy and Orphan Drug designations.

The evidence base is small. The open-label, multicenter trial enrolled 32 adults who had at least two surgeries in the prior year, including one scheduled at entry. Efficacy was judged in the 26 who had three or more. The 27% figure carries a 95% confidence interval of 12% to 48%, according to Inovio, and no comparison group received a placebo.

Safety findings were mostly mild, and Inovio reported that most treatment-related reactions were grade 1 or 2 and short-lived, with no common grade 4 or 5 events. The most common adverse reactions were injection site reactions, headache, fatigue, hypertension, nausea and dizziness. Electroporation can cause brief muscle contractions, temporary bleeding and vasovagal reactions, so patients are monitored for at least 30 minutes after an injection. Inovio says all 32 participants completed the study.

The company expects orders and shipments to begin in about 60 days, with full commercial launch in the first quarter of 2027, and it did not disclose a price. A draft confirmatory protocol is due to the agency by the end of 2026, with a final protocol targeted for mid-2027. The FDA’s list of approved cellular and gene therapy products, current as of Oct. 30, already includes Voceryq. Whether the 27% complete-response rate holds up in the larger confirmatory trial is the open question that will decide whether the approval converts to a traditional one.

This article was produced with the assistance of AI and reviewed by Morning Overview editors prior to publication.

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