The US Food and Drug Administration (FDA) has granted accelerated approval to Voceryq (doruxapapogene ralaplasmid and lalikinogene sifuplasmid-gxfd) for treating adults with recurrent respiratory papillomatosis (RRP), a rare disease caused by human papillomavirus (HPV) that results in the formation of small, wart-like growths in the airways.
Developed by Inovio Pharmaceuticals, Voceryq is the first plasmid DNA-based immunotherapy to receive approval in the US. The treatment offers a new therapeutic option for patients with limited treatment choices by targeting the root cause of RRP and potentially reducing the need for repeated surgical interventions.
“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 FDA’s Center for Biologics Evaluation and Research (CBER).
He described the approval as “a meaningful scientific breakthrough: a plasmid DNA-based immunotherapy that harnesses the body’s own immune system to fight the underlying cause of RRP”.
For patients, he added, the treatment could mean fewer surgeries, fewer disruptions and more time living their lives on their own terms.
Dr Jacqueline Shea, President and CEO of Inovio Pharmaceuticals, said, “Voceryq is designed to deliver what we understand matters most to patients, which is the reduction of the surgical burden caused by RRP.”
The FDA’s accelerated approval is supported by clinical trial data showing that Voceryq can reduce the frequency of surgical procedures required to remove growths in adults with RRP. The reduction in surgical interventions was considered an intermediate clinical endpoint reasonably likely to predict clinical benefit.
Under the FDA’s accelerated approval pathway, drugs and biologics intended to treat serious or life-threatening conditions may be approved based on their effect on a surrogate or intermediate clinical endpoint reasonably likely to predict clinical benefit. Manufacturers are required to conduct post-approval clinical trials to verify and describe the treatment’s clinical benefit.
Voceryq is approved for use in combination with CELLECTRA 5PSP, Inovio’s proprietary DNA medicine delivery device.
Recurrent respiratory papillomatosis
RRP is a rare, chronic disease in which wart-like growths, known as papillomas, develop in the respiratory tract due to infection with HPV types 6 and/or 11. The growths most commonly affect the larynx (voice box), particularly the vocal cords.
Symptoms can include hoarseness, chronic cough and changes in voice. In severe cases, the growths can spread to other parts of the respiratory tract and obstruct the airways, making breathing difficult.
Although surgery can remove the growths, there is currently no cure for RRP. The virus can persist in the affected tissue even after the papillomas are removed, meaning patients often require repeated surgical procedures over several years. In some cases, the disease also carries a risk of becoming cancerous over time.
How Voceryq works
Voceryq is a recombinant plasmid DNA-based immunotherapy that delivers two plasmids (small pieces of DNA originally derived from bacteria) into the body’s cells. One contains the genetic instructions for specific HPV proteins, while the other contains an immune response-enhancing molecule called IL-12.
The therapy is designed to stimulate the body’s immune system to recognise and attack cells infected with HPV types 6 and 11, which are responsible for RRP.
Voceryq is administered through intramuscular injection using the CELLECTRA 5PSP device. The device delivers the treatment and facilitates electroporation, a technique that uses brief electrical pulses to create temporary, reversible pores in cell membranes, enabling the plasmid DNA to enter the body’s cells.
Clinical trial findings
The safety and effectiveness of Voceryq were evaluated in an open-label, multicentre clinical trial involving 32 adults diagnosed with RRP.
Eligible participants had undergone at least two surgeries to remove laryngotracheal papillomas in the 12 months before receiving Voceryq, including a scheduled surgery at trial entry.
Among the 26 patients who had undergone at least three baseline surgeries, seven (27%) required no further surgical intervention during the first year after treatment.
The FDA had previously granted Voceryq Breakthrough Therapy and Orphan Drug designations.
Inovio expects Voceryq to be available for ordering and shipment in approximately 60 days, with full field deployment anticipated in the first quarter of 2027.
