FDA Approves Airway Immunotherapy for Rare Disease

The United States Food and Drug Administration granted accelerated approval on 30 October 2026 to Voceryq, an airway immunotherapy for adults with recurrent respiratory papillomatosis, a rare disease in which wart-like growths form in the airways. The approval is the first in the United States for a plasmid DNA-based immunotherapy, according to the FDA.

Recurrent respiratory papillomatosis is caused by human papillomavirus types 6 and 11, and the growths, called papillomas, most often form around the larynx and the vocal cords. Patients may develop hoarseness, a chronic cough and changes to the voice, and severe disease can spread through the respiratory tract and make breathing difficult. Surgery can remove the growths, but the virus persists in the tissue after removal, so patients often need repeated procedures over many years. In some cases, the disease carries a risk of becoming cancerous over time, according to the FDA.

Karim Mikhail, director of the FDA Center for Biologics Evaluation and Research, said in a statement: "Adults living with recurrent respiratory papillomatosis often face an exhausting and relentless cycle of repeated surgeries, managing their disease, but never truly escaping it." Mikhail said the approval could mean fewer surgeries, fewer disruptions and more time for patients to live their lives on their own terms.

Voceryq is designed to attack the underlying cause rather than only removing the growths after they appear. The therapy delivers plasmids into the body cells: a plasmid carrying genetic instructions for specific HPV proteins, and a plasmid carrying instructions for IL-12, a molecule that enhances the immune response. Together, they are intended to prompt the immune system to attack cells infected by the virus types that cause the disease. The therapy is given by intramuscular injection using the CELLECTRA 5PSP device, which uses brief electrical pulses to temporarily open pores in cell membranes so the plasmids can enter the cells, a technique called electroporation.

What the trial showed

The safety and effectiveness of Voceryq were evaluated in an open-label, multicentre clinical trial in 32 adults with the disease, according to the FDA. Eligible patients had undergone at least two surgeries to remove laryngotracheal papillomas in the year before treatment, including a scheduled surgery at trial entry. Seven of 26 patients who had at least three baseline surgeries needed no surgical intervention in the year after treatment, a result of 27 per cent in that group.

Across the full trial group, 81 per cent of patients needed fewer surgeries in the year after treatment than in the year before, according to Pharmacy Times. Investigators also observed HPV-specific T cells in peripheral blood, and T-cell receptor sequencing confirmed that newly emerged T-cell clones trafficked to papilloma tissue, as reported in the Nature Communications paper on the phase 1/2 study. Patients in the pivotal trial received four doses.

The most commonly reported adverse reactions were injection site reactions, headache, fatigue, hypertension, nausea and dizziness, according to the FDA. Adverse effects linked to electroporation may include brief muscle contractions or tetany, temporary bleeding at the administration site, anxiety, vasovagal reactions including lightheadedness and dizziness, transient changes in creatine phosphokinase, skin redness at the injection site and pain at the injection site. The agency said the therapy received breakthrough therapy and orphan drug designations.

What accelerated approval means

Accelerated approval allows the FDA to approve a drug or biologic for a serious condition based on an intermediate clinical endpoint that is reasonably likely to predict clinical benefit. In this case, the endpoint was a reduction in the frequency of surgical procedures required to remove growths. The manufacturer must conduct post-approval clinical trials to verify and describe the clinical benefit, and continued approval may depend on those results, according to the FDA.

Until recently, management of the disease relied entirely on surgery. In August 2025, the FDA approved zopapogene imadenovec, sold as Papzimeos, as a therapy for adults with the disease, according to Pharmacy Times. That treatment uses a nonreplicating adenoviral vector, making Voceryq mechanistically distinct because it is built on plasmid DNA rather than a viral vector. Confirmatory trial results will ultimately define the place of Voceryq in therapy alongside surgical management and Papzimeos.

For patients, the practical question is whether fewer operations translate into lasting control of a disease that returns again and again. The trial data point in that direction for a substantial share of participants, but the numbers come from a small study, and the confirmatory work required under accelerated approval will test whether the benefit holds in a larger group over a longer period. The FDA granted the approval to Inovio Pharmaceuticals.

Other recent health reporting on this site examined a hormone therapy heart study and a cancer immunotherapy diet study, both of which concern different conditions.