FDA clears first DNA immunotherapy for rare airway disease RRP

The FDA granted accelerated approval to Voceryq on 30 October 2026 for adults with recurrent respiratory papillomatosis (RRP), a rare disease in which HPV causes wart-like growths in the airway. Made by INOVIO, it is the first plasmid DNA immunotherapy approved in the US.

The approval is based on a single open-label trial of 32 adults. In the 26 people who had needed three or more operations in the year before treatment, 7 (27%) needed no surgery in the following 12 months. Because the approval is accelerated, INOVIO must run a confirmatory trial to show the benefit holds.

Voceryq is the second immunotherapy approved for RRP in the US. Precigen's Papzimeos was approved in 2025, and the two carry the same list price.

What the FDA approved

According to the FDA and INOVIO:

Accelerated approval lets the FDA approve treatments for serious conditions based on an effect on a surrogate or intermediate endpoint that is "reasonably likely to predict clinical benefit". Here that endpoint was the need for surgery.

What RRP is

The FDA describes RRP as a rare, chronic disease caused by HPV types 6 and 11. Papillomas grow in the airway, most often around the voice box and vocal cords. They cause hoarseness, a chronic cough and voice changes. In severe cases they spread down the airway and make breathing difficult.

Surgery removes the growths but does not cure the disease, because the virus stays in the tissue. Many people need repeated operations for years, and each one risks scarring the larynx. In some cases RRP can turn cancerous.

INOVIO estimates from US insurance claims that about 25,000 adults in the US have RRP, according to MedCity News.

How Voceryq works

Voceryq delivers two plasmids, small circles of DNA, into muscle cells. One carries instructions for HPV-6 and HPV-11 proteins. The other carries instructions for IL-12, a signalling molecule that boosts the immune response. The aim is to train the immune system to attack cells infected with these HPV types.

The CELLECTRA device uses electroporation: brief electrical pulses that open temporary pores in cell membranes so the DNA can enter. The full course is four doses.

The trial

The RRP-001 study enrolled 32 adults who had needed two or more surgeries in the previous 12 months, including one scheduled at entry. According to INOVIO:

• 26 of the 32 had needed three or more surgeries in the year before treatment, and formed the efficacy population
• their median number of surgeries in that year was 4.5 (range 3 to 8)
• 7 of 26 (27%; 95% confidence interval 12% to 48%) had a complete response, meaning no surgery in the 12 months after treatment
• all 32 completed the study

Results were published in Nature Communications and The Laryngoscope.

How to read the result

There are three caveats.

First, the trial had no control group. INOVIO argues that going a full year without surgery would not be expected from the natural course of the disease in people with this history. That is plausible but harder to judge without a comparison arm.

Second, the confidence interval is wide, from 12% to 48%, because the trial was small.

Third, most patients still needed surgery. About three in four did not reach a complete response. INOVIO's announcement does not give the average reduction in surgeries for the rest, so it is unclear how much benefit they gained.

MedCity News also notes that patients needed debulking surgery to remove papillomas before treatment, as with Papzimeos. Voceryq reduces the need for surgery; it does not replace it.

Safety

The most common side effects were injection site reactions, headache, fatigue, high blood pressure, nausea and dizziness. INOVIO says most treatment-related reactions were mild or moderate and short-lived, with no grade 4 or 5 common adverse events.

The FDA lists effects linked to electroporation, including brief muscle contractions, bleeding at the injection site, anxiety, light-headedness or fainting, changes in the muscle enzyme creatine phosphokinase, and redness and pain where the injection was given.

The competition

Papzimeos uses a gorilla adenovirus vector to deliver HPV-6 and HPV-11 antigens and received a full FDA approval in 2025. Voceryq has an accelerated approval, which is less secure.

INOVIO's chief executive, Jacqueline Shea, told investors that Voceryq does not require surgery before the third and fourth doses, unlike Papzimeos when papillomas are visible. "We designed the trial this way because every surgery matters to patients," she said, according to MedCity News. The two trials are different, so their response rates should not be compared directly.

Price

INOVIO set a US list price of $115,000 per dose, or $460,000 for the four-dose course, according to MedCity News. The company said this is in line with Papzimeos. INOVIO expects to ship Voceryq in about 60 days.

UK and EU access

Voceryq is not licensed in the UK or the EU. MedCity News reports that INOVIO is looking at seeking approvals in Europe, the UK and other markets. No timeline has been given. In the UK, RRP is managed by ear, nose and throat surgeons, and treatment remains surgical, sometimes with off-label drugs.

What happens next

Continued approval depends on a confirmatory study that verifies the clinical benefit. INOVIO says the FDA requires it to submit a draft protocol by the end of 2026, with the aim of agreeing a final protocol by mid-2027. The FDA's approval letter, published alongside the decision, sets out the study requirements and deadlines.

If that study does not confirm a benefit, the FDA can withdraw the approval. INOVIO also plans a full commercial rollout in the first quarter of 2027.

This article explains published research and isn't medical advice. Talk to your own doctor or specialist team before changing any treatment.