FDA clears first DNA-based therapy for rare throat disease, aiming to spare patients repeat surgery

The Food and Drug Administration has granted accelerated approval to the first DNA-based immunotherapy for recurrent respiratory papillomatosis, a rare disease that has forced many patients into a lifelong cycle of throat surgery just to keep breathing and speaking normally. The approval, announced by the agency on Oct. 30, gives adults with the condition a non-surgical option for the first time anywhere in this drug class, rather than the repeated operations that have long been the only way to manage it.

The therapy, developed by Inovio Pharmaceuticals and sold under the brand name Voceryq, was cleared under the FDA's accelerated approval pathway, which allows faster authorization of treatments for serious diseases based on results considered likely, though not yet proven, to predict lasting clinical benefit. Formerly tested under the name INO-3107, it is the first plasmid DNA-based medicine ever approved for use in the United States, a distinction the agency's Center for Biologics Evaluation and Research said validates an entire class of experimental genetic therapies still working their way through development.

A disease with no alternative to the scalpel

Recurrent respiratory papillomatosis, known as RRP, is caused almost entirely by two low-risk strains of human papillomavirus, HPV-6 and HPV-11, the same types responsible for most genital warts. The virus produces wart-like growths, called papillomas, in the airway, most often clustered around the vocal cords. Left alone, the growths multiply and can obstruct breathing. According to the National Institute on Deafness and Other Communication Disorders, there is no cure, and surgery to shave away the growths with a laser or microdebrider has been the standard of care for decades. Because the virus persists in surrounding tissue even after an operation, papillomas typically regrow, and patients can require repeat procedures every few weeks to every few months for years on end.

Inovio, drawing on an analysis of insurance claims data, estimates that at least 25,000 people in the United States currently live with RRP, with new cases diagnosed every year; the patient advocacy group the RRP Foundation separately puts the number of active cases at roughly 20,000. Adults can develop the disease from sexual transmission, while children most often acquire it from a mother during childbirth, a form that tends to run a more aggressive course. Vaccination against HPV has been associated with fewer new juvenile cases in the United States in the years since it became routine, though it offers no benefit to people already infected.

Targeting the virus rather than the symptom

Voceryq works differently from surgery or from the only other approved RRP drug, Precigen's Papzimeos, which uses a modified virus to carry its payload. Voceryq instead delivers two small loops of synthetic DNA, called plasmids, directly into muscle tissue using an electrical-pulse device called CELLECTRA that briefly opens pores in cell membranes. One plasmid carries genetic instructions for proteins from HPV-6 and HPV-11; the other encodes an immune-signaling molecule, interleukin-12, meant to amplify the response. Together they are designed to train the immune system to recognize and attack cells already infected with the virus, addressing what the FDA's press release called the disease's underlying cause rather than only the growths it produces.

The approval rests on a 32-patient, open-label trial known as RRP-001, in which all participants had needed at least two surgeries in the prior year. Among the 26 patients who had required three or more surgeries, seven, or 27 percent, went a full year afterward without needing any surgical excision at all, a result the company and FDA regard as unlikely to occur naturally in untreated disease. The dosing regimen runs nine weeks and follows an initial surgical debulking of visible growths; no further surgery is required during that window. Results from the trial and a longer-term follow-up study were published in Nature Communications and, this past August, in the journal Laryngoscope. All 32 patients completed the study, and the most common side effects — injection-site reactions, headache, fatigue, elevated blood pressure, nausea and dizziness — were mostly mild and temporary, with no severe adverse events reported.

'Patient voices matter'

The approval drew swift praise from the small community of specialists and patients who treat and live with RRP, a disease serious enough to occasionally require a tracheotomy to keep the airway open, yet obscure enough that it has drawn little research investment relative to its toll on quality of life. Dr. Jacqueline Shea, Inovio's chief executive, said in the company's announcement that the therapy was built around what mattered most to patients. Dr. Simon Best, an associate professor of otolaryngology at Johns Hopkins University and a lead investigator on the trial, said the approval was "an important step in improving the quality of life for RRP patients as every surgery carries the risk of laryngeal injury," adding that he believes treatment should move quickly away from surgery once the disease is diagnosed.

"This new FDA-approved therapy brings us an important step closer to ensuring every RRP patient has access to a non-surgical treatment option that works for them. In reaching this milestone, we have shown that patient voices matter in the drug development process," said Kim McClellan, president of the RRP Foundation.

For patients, the stakes of repeated surgery go beyond inconvenience. Each operation carries a risk of scarring the vocal cords, which can permanently weaken or alter the voice, and the cumulative financial and emotional cost of procedures spaced only weeks apart has been a defining feature of life with the disease. Because RRP concentrates its damage on the larynx, it disproportionately affects people whose livelihoods or daily lives depend on speaking clearly, on top of the basic risk that a growth left unchecked can choke off the airway entirely.

A conditional approval, with a confirmatory trial still to come

Because Voceryq was cleared under the accelerated pathway, its continued approval depends on Inovio completing a follow-up study that verifies the surgery-reduction benefit holds up over time; the FDA has told the company to submit a draft protocol for that study by the end of this year, with a final design expected by the middle of 2027. Inovio has said it expects to begin shipping the therapy within about 60 days, with a fuller commercial rollout, supported by the contract firm Syneos Health, planned for the first quarter of 2027. The company also received Orphan Drug and Breakthrough Therapy designations for the treatment, both of which are reserved for medicines addressing rare or serious unmet needs.

Financial markets treated the decision as a turning point for Inovio, a company that has struggled for years to bring a product to market; shares surged in after-hours trading following the announcement, while shares of Precigen, whose Papzimeos now faces its first direct competitor, fell. For the roughly 25,000 Americans living with RRP, the more immediate question is simpler: whether a treatment aimed at the virus itself can finally loosen a disease that, until now, only a scalpel could hold back, and whether that benefit persists once more patients outside a small clinical trial have had the chance to try it. The approval covers adults only; the disease's often more severe juvenile-onset form, typically acquired at birth, is not included in the new indication, and the HPV vaccine remains the only tool aimed at preventing new cases altogether.