After an initial FDA scare that threatened its path for an accelerated approval, Inovio’s Voceryq, the first plasmid DNA-based immunotherapy, has earned a go-ahead to treat a rare HPV-driven disease.
Friday’s approval allows Voceryq to reduce the requency of surgery in adults with recurrent respiratory papillomatosis (RRP), in which HPV infection leads to small, wart-like growths in the airways and on the vocal cords.
The accelerated approval follows a rocky start. When accepting Inovio’s application in March, the FDA indicated in a preliminary conclusion that the company had not submitted adequate information to pursue this expedited pathway. The hiccup underscored a broader, tightened regulatory approach toward rare disease under then-FDA Commissioner Marty Makary, M.D.
In an Oct. 30 statement, Karim Mikhail, the FDA’s director of the Center for Biologics Evaluation and Research since mid-May, hailed Voceryq as “a meaningful scientific breakthrough,” and a therapy that “could mean fewer surgeries, fewer disruptions, and more time living their lives on their own terms.”
“Adults living with recurrent respiratory papillomatosis often face an exhausting and relentless cycle of repeated surgeries, managing their disease, but never truly escaping it,” said Karim Mikhail, director of the Center for Biologics Evaluation and Research. “Today’s approval represents a meaningful scientific breakthrough: a plasmid DNA-based immunotherapy that harnesses the body’s own immune system to fight the underlying cause of RRP.”
Before Voceryq, patients with RRP faced repeated surgeries to relieve symptoms such as difficulty speaking, swallowing and breathing. Each procedure carries the risk of permanent damage to the vocal cords or airway, Inovio Chief Medical Officer Michael Sumner explained during a Nov. 2 investor call.
Voceryq is composed of two DNA plasmids, one encoding for the proteins specific to the HPV6 and 11 viruses behind RRP, and the other for human IL-12 to enhance the immune response. Much like a vaccine, Voceryq enables the muscle cells to produce the HPV viral proteins, giving the immune system a target to recognize.
In a multicenter clinical trial involving 32 adults who had undergone at least two surgeries in the year before treatment, seven of the 26 patients (27%) who had undergone at least three surgeries during that period required no additional surgery in the first year after treatment.
The drug is given using a proprietary hand-held device, which enables targeted, localized delivery, Sumner noted. Voceryq can be stored in standard refrigeration with a three-year shelf life.
“These straightforward distribution requirements should facilitate flexibility in the administration location, including the doctor's office,” Sumner said.
Based on its analysis of claims data, Inovio estimates that at least 25,000 people in the U.S. are living with RRP. With patients seeking alternatives to surgery, “the vast majority” of the market remains available to Voceryq, Inovio CEO Jacqueline Shea, Ph.D., said during a Nov. 2 investor call.
“The KOL community is extremely supportive of starting immunotherapy as soon as possible after diagnosis,” Shea said, pointing to a position statement recently published by the Recurrent Respiratory Papillomatosis Foundation.
Under the accelerated approval pathway, Inovio is working on a confirmatory trial plan with a draft protocol expected for FDA review before the end of the year and final alignment by mid-2027, Sumner said.
“We have agreed with the agency that the study should enroll at least 50 adult and pediatric patients with RRP requiring three or more surgical interventions in the 12 months prior to treatment with Voceryq,” he said on the call. “We expect the study to evaluate the number of surgical interventions required over at least three years following completion of treatment, and compare this to an appropriate external control group or scientifically justified threshold. We may also explore continued treatment in patients without a sustained complete response as part of the study.”
Following Friday’s approval, Inovio’s shares nearly tripled in value, reaching about $1.80 apiece as of Monday morning.