FDA Grants Accelerated Approval for Inovio’s RRP Immunotherapy

Rare Daily Staff

The U.S. Food and Drug Administration granted accelerated approval to Inovio’s Voceryq, a DNA-based immunotherapy designed to reduce the frequency of surgery in adults with recurrent respiratory papillomatosis, a rare disease that can require repeated operations to remove growths in the airway.

Inovio described Voceryq as the first DNA medicine approved by the FDA, making the decision a milestone for both its RRP program and its broader technology platform.

The approval was based on patients who went 12 months without requiring surgery after treatment. Continued approval may depend on a confirmatory study verifying the therapy’s clinical benefit.

Recurrent respiratory papillomatosis, or RRP, is a rare, potentially life-threatening disease of the upper and lower respiratory tract caused by chronic infection with human papillomavirus type 6 or type 11. It can lead to severe voice problems, airway obstruction and repeated bouts of pneumonia. Treatment has mainly involved repeated surgeries, which do not address the underlying cause and can carry significant risks and burdens for patients and the healthcare system.

Voceryq is a recombinant plasmid DNA-based immunotherapy that delivers two small pieces of DNA into the body’s cells. One carries genetic instructions for specific HPV proteins; the other carries instructions for an immune response-enhancing molecule called IL-12. The therapy is designed to stimulate the immune system to recognize and attack cells infected by the HPV types that cause RRP.

Voceryq is administered by intramuscular injection in conjunction with the Cellectra 5PSP device, which facilitates electroporation. This technique uses brief electrical pulses to temporarily create pores in cell membranes, allowing the DNA to enter cells.

The FDA’s decision was supported by the company’s RRP-001 study, which enrolled 32 adults who had undergone at least two surgeries in the year before treatment.

The efficacy analysis included 26 participants who had required three or more surgeries during that period. Those patients had undergone a median of 4.5 surgical procedures in the preceding year, with individual totals ranging from three to eight.

Seven of the 26 patients, or 27 percent, required no surgical removal of papillomas during 12 months of follow-up, meeting the study’s definition of a complete response. The 95 percent confidence interval ranged from 12 percent to 48 percent, reflecting uncertainty around the response estimate in the small population.

The result does not establish that Voceryq eliminates RRP or that patients will remain free of surgery beyond the follow-up period. Although the efficacy analysis focused on patients with at least three prior surgeries, the approved indication described by Inovio covers adults with RRP without specifying a minimum number of previous procedures.

The most common adverse reactions, occurring in more than 5 percent of patients, included injection-site reactions, headache, fatigue, hypertension, nausea and dizziness, according to Inovio. Most treatment-related reactions were mild or moderate and transient. All 32 participants completed the study.

Inovio said the FDA requires it to submit a draft protocol for a confirmatory study by the end of 2026, with agreement on a final protocol expected by mid-2027.