FDA Approves Voceryq, the First Plasmid DNA Immunotherapy, for a Rare Airway Disease
Inovio's treatment for recurrent respiratory papillomatosis is cleared under accelerated approval, giving adults a new option beyond repeated surgery.
On October 30, the U.S. Food and Drug Administration approved Voceryq, made by Inovio Pharmaceuticals, for adults with recurrent respiratory papillomatosis (RRP). The agency calls it the first plasmid DNA-based immunotherapy approved in the United States, and the approval was granted on an accelerated basis.
"Today's approval represents a meaningful scientific breakthrough." — Karim Mikhail, Director of the FDA's Center for Biologics Evaluation and Research
A condition with no cure
RRP is a rare, chronic disease in which HPV types 6 and 11 cause wart-like growths in the airways, most often around the larynx and vocal cords. People can face hoarseness, a chronic cough and, in severe cases, trouble breathing. Surgery removes the growths, but the virus stays in tissue, so many patients need repeated procedures over years.
How Voceryq works
The treatment delivers two plasmids, small loops of DNA, into the body's cells. One carries instructions for HPV proteins; the other carries IL-12, a molecule that boosts the immune response. The aim is to help the immune system target cells infected with those two HPV types. It is injected into muscle using a device that applies brief electrical pulses to help the DNA enter cells.
What the trial showed
- The open-label study enrolled 32 adults who had needed at least two airway surgeries in the prior year
- Among 26 patients who had three or more surgeries at baseline, 7 (27%) needed no surgical intervention in the year after treatment
- Common side effects included injection-site reactions, headache, fatigue, high blood pressure, nausea and dizziness
What happens next
Accelerated approval rests on reduced surgery frequency, a measure the FDA considers reasonably likely to predict real clinical benefit. The company must now run follow-up trials to confirm that benefit. The product also carried the FDA's Breakthrough Therapy and Orphan Drug designations.
The FDA said the approval could mean fewer surgeries and fewer disruptions for patients, a meaningful step for a community that has had few options beyond the operating room.
