FDA Grants Accelerated Approval to Inovio's Voceryq, First Plasmid DNA Immunotherapy, for Recurrent Respiratory Papillomatosis

The U.S. Food and Drug Administration on Friday, Oct. 30, granted accelerated approval to Voceryq, a plasmid DNA-based immunotherapy from Inovio Pharmaceuticals, for adults with recurrent respiratory papillomatosis (RRP). It is the first plasmid DNA immunotherapy approved in the United States.

RRP is a rare, chronic disease. HPV types 6 and 11 cause wart-like growths, called papillomas, in the airway, most often on the larynx and vocal cords. They can cause hoarseness, chronic cough and, in severe cases, breathing difficulty. The FDA notes that in some cases the disease can become cancerous over time.

There is no cure. Surgery removes the growths, but the virus stays in the tissue, so patients often return for repeat procedures over many years.

What the drug is and how it works

Voceryq (doruxapapogene ralaplasmid and lalikinogene sifuplasmid-gxfd), previously called INO-3107, delivers two plasmids. One carries instructions for HPV-6 and HPV-11 proteins. The other carries instructions for interleukin-12, an immune-stimulating molecule.

It is given by intramuscular injection with Inovio's CELLECTRA 5PSP device, which uses brief electrical pulses to help the DNA enter cells. The FDA approved the drug and device together.

"Today's approval represents a meaningful scientific breakthrough," said Karim Mikhail, director of the FDA's Center for Biologics Evaluation and Research. "For patients, that could mean fewer surgeries, fewer disruptions, and more time living their lives on their own terms."

The trial behind the approval

The approval rests on RRP-001, an open-label, multicenter study of 32 adults who had at least two papilloma surgeries in the prior 12 months. All 32 completed the study, according to Inovio. Patients had standard surgical debulking first, then a 9-week dosing regimen.

The efficacy population was narrower: 26 patients with three or more surgeries in the year before enrollment, with a median of 4.5 (range 3 to 8). In that group, 7 of 26, or 27%, had a complete response, defined as no surgery through 12 months of follow-up. The 95% confidence interval runs from 12% to 48%.

That means roughly three in four patients in the efficacy group did not hit that mark. The trial had no control arm. Inovio says complete responses of this kind are not expected in the untreated natural history of the disease.

The FDA based its decision on reduced surgery frequency, which it calls an intermediate endpoint "reasonably likely to predict clinical benefit." That is the standard for accelerated approval. Inovio must run confirmatory studies, and the agency says continued approval can depend on verifying the benefit.

Simon Best, an associate professor of otolaryngology at Johns Hopkins and a lead investigator on RRP-001, said every surgery carries a risk of laryngeal injury. "I believe it is in the best interest of RRP patients that the standard of care quickly transition away from surgical interventions to immunotherapy after RRP is diagnosed," he said.

A second option in just over a year

Voceryq is the second approved RRP therapy. The FDA approved Papzimeos (zopapogene imadenovec-drba), an adenoviral vector-based immunotherapy, a little more than a year ago.

Tech Times, citing its own figures, put the U.S. population with active RRP at roughly 14,000, with about 1,800 new adult cases diagnosed each year.

Inovio's timeline and stock

Inovio (NASDAQ: INO) said it expects Voceryq to be available to order and ship in about 60 days, with a full commercial launch in the first quarter of 2027. CEO Jacqueline Shea called it the first FDA-approved DNA medicine and said it "has the potential to become a new standard of care."

The market reacted fast. Dow Jones Newswires reported that Inovio shares more than tripled in after-hours trading Friday to $2.17. That was an after-hours move, not a regular-session price. U.S. markets are closed for the weekend, and regular trading resumes Monday, Nov. 2.

Inovio will host a conference call at 8:00 AM ET that day.

The confirmatory work remains open. The FDA has not publicly detailed the design or timeline of those post-approval trials in its announcement. Until they report, the 27% complete-response figure from a single-arm trial of 26 efficacy patients is the evidence behind the approval.