FDA Approves the First DNA-Based Immunotherapy, on a 32-Patient Trial
Inovio's Voceryq is the first plasmid DNA-based immunotherapy cleared in the U.S., winning accelerated approval for a rare airway disease on a 32-patient study. The confirmatory trial is still ahead.
The U.S. Food and Drug Administration on October 30, 2026 granted accelerated approval to Inovio Pharmaceuticals' Voceryq for adults with recurrent respiratory papillomatosis. The agency called the decision the first for a plasmid DNA-based immunotherapy in the United States. Voceryq (doruxapapogene ralaplasmid and lalikinogene sifuplasmid-gxfd) is indicated to reduce the frequency of surgical papilloma excisions. RRP is a rare, chronic disease in which HPV types 6 and/or 11 cause wart-like growths in the airway, where repeated surgery has long been the standard of care and there has been no cure.
The scientific first is real, and so is the modest evidence behind it. Accelerated approval rests on an intermediate endpoint, a single open-label trial, and a confirmatory protocol that has not yet been finalized. This is Inovio's first approved product. The questions that follow are whether the reduction in surgery holds up in a confirmatory trial, and whether a company that has never commercialized a drug can execute a rare-disease launch on its stated timetable.
What the 32-patient study showed
Voceryq is given by intramuscular injection using Inovio's CELLECTRA 5PSP device, which uses brief electrical pulses (electroporation) to deliver the DNA. Accelerated approval was based on the intermediate endpoint of reduced surgery frequency, and continued approval may hinge on a confirmatory study.
The RRP-001 trial enrolled 32 adult patients who had two or more surgeries in the prior year. Efficacy was assessed in 26 patients who had three or more baseline surgeries. In that group, the median number of surgeries in the prior 12 months was 4.5, with a range of 3 to 8. Seven of 26 patients (27%, 95% confidence interval 12% to 48%) had a complete response, defined as no surgical excision for 12 months after treatment.
The label is not a claim of cure. It is a claim about fewer operations. In a disease built around repeat excision, avoiding surgery for a full year after treatment is the endpoint that mattered, and it was observed in 7 of 26 patients. The confidence interval is wide enough that the true complete-response rate could sit nearer 12% or nearer 48%. That is what a 26-patient efficacy analysis can and cannot settle. It was enough for accelerated approval. It leaves a large gap for a confirmatory study to fill.
The most common adverse reactions were injection site reactions, headache, fatigue, hypertension, nausea, and dizziness.
A confirmatory protocol is still due
Inovio must submit a draft confirmatory-study protocol by the end of 2026, with a final protocol expected by mid-2027. The FDA granted Voceryq Breakthrough Therapy and Orphan Drug designations, and the European Commission granted orphan designation.
The designations mark a rare disease with no cure and a surgical standard of care, but they do not replace the confirmatory study. Reduced surgery frequency was accepted as the basis for the U.S. decision, and continued approval may hinge on the later trial. Until a final protocol exists, that trial is a requirement on the calendar rather than a fully specified public design. The dates that are public are a draft by the end of 2026 and a final protocol by mid-2027.
First product, first commercial test
Inovio expects Voceryq to be available to order and ship in about 60 days, with a full commercial launch in the first quarter of 2027, supported by Syneos Health field teams. The company estimates at least 25,000 prevalent RRP patients in the United States.
A 60-day ship window and a first-quarter 2027 launch would be a tight timetable for any first commercial product, and tighter still for a plasmid DNA immunotherapy that must be delivered with an electroporation device by a company that has not sold a drug before. The 25,000 figure is a prevalence estimate, not a forecast of how many patients will be treated after launch.
Shares more than tripled in after-hours trading to $2.17 following the approval. Inovio's earlier COVID-19 vaccine work did not produce an approved product. October 30 gave the company a U.S. label it has never held, for the first plasmid DNA-based immunotherapy cleared in the United States. Whether 7 complete responses in 26 patients can be confirmed, and whether Inovio and Syneos can find and treat RRP patients on the company's stated schedule, is the work still ahead.
