FDA Grants Accelerated Approval to First Plasmid DNA Immunotherapy for Recurrent Respiratory Papillomatosis
Inovio's Voceryq gives adults with a rare, HPV-driven airway disease a new option aimed at cutting repeat surgeries.
The U.S. Food and Drug Administration granted accelerated approval on October 30 to Voceryq, made by Inovio Pharmaceuticals, for adults with recurrent respiratory papillomatosis (RRP). The FDA calls it the first plasmid DNA-based immunotherapy approved for the condition.
The condition
RRP is a rare, long-lasting disease in which HPV types 6 and 11 cause wart-like growths in the airway, most often around the voice box. Symptoms include hoarseness and chronic cough, and severe cases can make breathing difficult. Surgery removes the growths, but the virus remains, so many patients need procedure after procedure.
How the treatment works
Voceryq is a two-part DNA immunotherapy. One plasmid carries instructions for proteins from the HPV types behind RRP, and the other carries IL-12, a molecule that boosts immune activity. Together they are meant to help the body's own immune system target infected cells. It is given as an intramuscular injection with a device that uses brief electrical pulses to help the DNA enter cells.
What the trial showed
- An open-label, multicenter trial enrolled 32 adults who had needed two or more airway surgeries in the prior year
- Among the 26 participants with at least three surgeries before treatment, 7 (27%) needed no surgery in the year after treatment
- Common side effects were injection-site reactions, headache, fatigue, high blood pressure, nausea and dizziness
The drug had Breakthrough Therapy and Orphan Drug designations. Karim Mikhail, director of the FDA's Center for Biologics Evaluation and Research, said patients could see "fewer surgeries, fewer disruptions."
What's next
Accelerated approval rests on an intermediate measure, reduced surgery frequency, that is considered reasonably likely to predict real clinical benefit. Inovio must complete follow-up trials to confirm that benefit. The trial was small and had no comparison group, so the confirmatory studies will matter. Patients should talk with their own doctors about whether treatment is appropriate.
