America approves its first plasmid DNA medicine, on condition that a further study confirms it works

ON OCTOBER 30TH America's Food and Drug Administration (FDA) approved Voceryq, a medicine made by Inovio Pharmaceuticals, for adults with recurrent respiratory papillomatosis (RRP). In this rare disease, two strains of human papillomavirus, HPV-6 and HPV-11, cause wart-like growths in the airway, most often on the vocal cords in the voice box. Surgeons can cut the growths out, but the virus stays in the surrounding tissue, so the growths return and many patients need repeated operations over many years. The FDA puts the number of new cases in America at about 1,000 a year.

Voceryq consists of small rings of DNA, first taken from bacteria, that are known as a plasmid. One of its two plasmids carries instructions for HPV proteins, and the other carries instructions for IL-12, a molecule that strengthens immune responses. The aim is for the patient's own cells to make these proteins, so the immune system learns to attack cells infected by the virus. The DNA is injected into muscle, and a device called CELLECTRA 5PSP then sends brief electrical pulses that open small, temporary pores in the cells so that the DNA can get in, a technique called electroporation. The FDA's release calls the decision "the first for a plasmid DNA-based immunotherapy in the U.S."

Normally the FDA approves a medicine once studies show it helps patients in a way that matters to them. Under a faster route, the agency may approve a medicine for a serious disease on an earlier measure that is "reasonably likely" to predict such benefit, provided the company keeps studying it. This route is called accelerated approval. The approval letter, signed by Vincent Amatrudo and Rosa Sherafat, two acting directors in the FDA's Center for Biologics Evaluation and Research (CBER), limits the medicine to reducing "the frequency of surgical papilloma excisions in adults with recurrent respiratory papillomatosis".

The evidence comes from one study, RRP-001, which enrolled 32 adults who had needed at least two operations in the previous year. Every participant received the medicine, so there was no comparison group of untreated patients against which to judge the results. The FDA measured effect in the 26 patients who had needed at least three operations in the year before treatment (a median of 4.5). Seven of those 26 (27%) needed no surgery at all in the year after treatment. Inovio reports that the true rate could plausibly lie between 12% and 48%. Its release quotes Simon Best, an associate professor of otolaryngology at Johns Hopkins University and a lead investigator on the study, as saying that "every surgery carries the risk of laryngeal injury".

The letter sets out the follow-up the FDA requires. A study run to check that the early result really reflects a benefit to patients is known as a confirmatory trial. Inovio's must enroll at least 50 adults and children who needed three or more operations in the previous year, and it must count their operations for at least three years after treatment. The results will be compared with "an appropriate external control group or scientifically justified threshold". A draft plan is due by December 30th 2026 and the study must be completed by December 30th 2031. The letter warns that if the study "fails to verify that clinical benefit is conferred" or is not conducted with due diligence, "we may withdraw this approval."

The FDA did not ask its panel of outside experts on cell and gene therapies to review the application. The letter explains that the evidence "did not raise concerns or controversial issues which would have benefitted from an advisory committee discussion." Karim Mikhail, the director of CBER, said in the agency's release that for patients the medicine "could mean fewer surgeries".

Voceryq is the second medicine approved for the disease. On August 14th 2025 the FDA approved Papzimeos, made by Precigen, which uses a modified virus rather than plasmids to train the immune system against the same two HPV strains. In its study, which also had no comparison group, 18 of 35 patients (51.4%) needed no surgery in the year after treatment. The FDA's release on Papzimeos does not describe that approval as accelerated.

Inovio expects the medicine to be available to order in about 60 days. The approval will stand on its present evidence only until the confirmatory trial reports, which the FDA's schedule places at the end of 2031.