FDA Approves Voceryq for Recurrent Respiratory Papillomatosis
The FDA has granted accelerated approval to Voceryq, the first plasmid DNA-based immunotherapy for adults with recurrent respiratory papillomatosis, a rare disease caused by HPV.
The US FDA has granted accelerated approval to Voceryq to reduce the frequency of surgical procedures in adults with recurrent respiratory papillomatosis. The treatment, developed by Inovio Pharmaceuticals, is the first authorisation of a plasmid DNA-based immunotherapy in the United States.
Karim Mikhail, Director of the FDA's Center for Biologics Evaluation and Research, highlighted the burden of the disease. "Adults living with recurrent respiratory papillomatosis often face an exhausting and relentless cycle of repeated surgeries, managing their disease, but never truly escaping it," he said.
Disease background
Recurrent respiratory papillomatosis is a rare, chronic disease caused by human papillomavirus types 6 and/or 11. The virus leads to wart-like growths forming in the airways, most often around the larynx. While surgery can remove these growths, there is no cure. The virus persists in the tissue, so patients often need repeated procedures over many years. In some cases, the condition carries a risk of becoming cancerous over time.
Treatment details
Voceryq is a plasmid DNA-based immunotherapy that targets the root cause of the disease. It delivers two plasmids: one encodes HPV-6 and HPV-11 antigens, and the other encodes the immune-enhancing cytokine interleukin-12. The treatment is administered via intramuscular injection using Inovio's proprietary CELLECTRA 5PSP Device. This device uses electroporation to facilitate the delivery of the DNA into cells. The FDA approved the drug and device together.
Clinical trial results
The safety and effectiveness of Voceryq were evaluated in an open-label, multicenter clinical trial called RRP-001. The trial enrolled 32 adults diagnosed with RRP who had at least two surgeries to remove laryngotracheal papillomas in the 12 months prior to treatment. All 32 participants completed the study, which had no control arm.
Before the first dose, patients underwent surgical debulking of visible papillomas. They then followed a 9-week dosing regimen with no additional surgeries required during that window. The efficacy analysis focused on 26 patients who had undergone three or more surgeries in the year before enrollment. This group had a median of 4.5 baseline surgeries.
| Trial Measure | Result in Efficacy Population (n=26) |
|---|---|
| Patients achieving complete response* | 7 (27%) |
| 95% Confidence Interval | 12% to 48% |
| Median surgeries in prior year | 4.5 |
| Range of prior surgeries | 3 to 8 |
*Complete response defined as no surgical intervention through 12 months of follow-up.
In this group, seven patients (27%) achieved a complete response, meaning they required no surgical intervention during the first year of follow-up. According to Inovio, complete responses of this kind are not expected in the untreated natural history of the disease. Complete clinical data were published in Nature Communications and The Laryngoscope.
Safety and future requirements
The therapy was well tolerated. The most common adverse reactions, with an incidence greater than 5%, included injection site reactions, headache, fatigue, hypertension, nausea, and dizziness. Potential effects from the electroporation process included brief muscle contractions, temporary bleeding at the site, anxiety, vasovagal reactions, and transient changes in creatine phosphokinase levels. Inovio reported most treatment-related reactions were grade 1 or 2 and transient. No grade 4 or 5 common adverse events occurred.
The label includes warnings for injection site reactions. Inovio advises monitoring patients for at least 30 minutes after each treatment. Clinicians should avoid using the device on the same side as an implanted cardioverter-defibrillator or pacemaker and avoid treatment sites with metal implants or skin abnormalities.
The FDA granted this accelerated approval based on the reduction in surgery frequency, an intermediate endpoint considered reasonably likely to predict clinical benefit. The approval comes with requirements for post-marketing study. The manufacturer must conduct post-approval clinical trials to verify and describe Voceryq’s clinical benefit. Inovio must submit a draft protocol for a confirmatory study by the end of 2026, with final protocol alignment expected by mid-2027. Continued approval may depend on the results of this confirmatory trial.
The FDA had previously granted Voceryq both Orphan Drug and Breakthrough Therapy designations. Under its development name, INO-3107, it also holds European Commission Orphan Drug designation. Inovio expects to make Voceryq available to order and ship in approximately 60 days.
