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The FDA granted accelerated approval to Voceryq, a plasmid DNA-based immunotherapy for adults with recurrent respiratory papillomatosis, to reduce the frequency of surgeries to remove airway growths. In a 32-patient open-label trial, 27% of a subgroup of 26 patients had no excisions during 12 months of follow-up; postmarketing trials must verify the treatment’s clinical benefit.
The U.S. Food and Drug Administration granted accelerated approval to Voceryq, a plasmid DNA-based immunotherapy intended to reduce how often adults with recurrent respiratory papillomatosis need surgery to remove airway growths. The approval gives patients with the rare, HPV-related disease a medical treatment option, while requiring postmarketing trials to verify and describe its clinical benefit.
The FDA based its decision on efficacy data from RRP-001, an open-label phase I/II trial involving 32 people who had undergone at least two surgeries to remove papillomas in the year before enrollment. Among the 26 patients with at least three surgeries in that period, seven—27%—had a complete response, defined as no surgical excisions during the 12 months after treatment. The result applies to that subgroup and follow-up period; it does not mean every treated patient avoided surgery.
Voceryq, whose generic name is doruxapapogene ralaplasmid-lalikinogene sifuplasmid, contains two plasmids carrying genetic coding for HPV proteins and interleukin 12. According to the report, patients’ healthy cells use that coding to stimulate immune responses against cells infected with HPV types 6 and 11, which cause recurrent respiratory papillomatosis. The regimen starts with surgery to remove visible papillomas, followed by four intramuscular doses given every three weeks.
The treatment is approved for use with the CELLECTRA 5PSP delivery device. The label warns of possible muscle contractions, temporary bleeding and vasovagal reactions. Common adverse events reported in trials included injection-site reactions, headache, fatigue, hypertension, nausea and dizziness. The available report does not provide detailed event rates or a comparison with a control group.
A New Option Alongside Repeat Surgery
Recurrent respiratory papillomatosis can require repeated operations because wart-like growths caused by HPV return in the airway, particularly around the larynx. In severe cases, growths may extend through the respiratory tract and make breathing difficult. Surgery can remove them, but it does not cure the disease, and repeated procedures can carry risks.
Investigator Simon Best of Johns Hopkins University said in a statement from manufacturer Inovio Pharmaceuticals that each operation carries a risk of laryngeal injury and described the approval as a step toward improving patients’ quality of life. The trial result offers a signal that some patients may go a year without excision after treatment, but it does not establish that surgery can be replaced for all patients. The regimen itself begins with surgical removal of visible growths.
The approval also adds to a small set of medical options for the condition. The FDA approved zopapogene imadenovec (Papzimeos) for RRP in 2025, described in the report as the first approved medical therapy for the disease. Voceryq is identified as the first FDA-approved recombinant plasmid DNA-based immunotherapy.
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How RRP Treatment Has Developed
Recurrent respiratory papillomatosis, or RRP, is associated with HPV and causes papillomas—wart-like growths—in the airways. The larynx is a common site. The disease can recur after removal, leaving patients in need of further procedures; there is no cure, according to the source report.
The FDA’s approval is accelerated, not a final resolution of questions about long-term clinical benefit. The decision relied on RRP-001, a small, open-label phase I/II trial. The report says further trials after approval are required to verify and describe benefit. Its account does not include trial results beyond the 12-month response measure or provide a head-to-head comparison with other treatments.
“The approval of Voceryq is an important step in improving the quality of life for RRP patients as every surgery carries the risk of laryngeal injury.”
— Simon Best, MD, a study investigator at Johns Hopkins University, in a statement from Inovio Pharmaceuticals
Questions for Confirmatory Trials
The FDA’s accelerated approval requires postmarketing studies to verify and describe Voceryq’s clinical benefit. The source report does not specify their design, enrollment targets, completion dates or what outcomes will be used. It also does not provide enough information to assess how durable responses may be beyond the reported 12 months.
The trial was open-label and involved 32 patients; the supplied results do not describe a control group or comparative effectiveness against surgery or other therapies. The 27% complete-response figure covers only the 26 participants who had at least three procedures in the prior year. How well the result applies to the broader RRP population, and which patients are most likely to benefit, remain unclear from the available information.
Follow-Up Evidence After Approval
Inovio and other parties involved in the required postmarketing trials will need to generate evidence for the FDA to review on the therapy’s clinical benefit. The source report does not give a timeline for those studies or say when results are expected. In the meantime, the approval makes Voceryq available as an option for eligible adults, with a treatment course that combines initial surgical debulking and four doses of the immunotherapy.
Further reporting will be needed to clarify how clinicians use the treatment in practice, how long any reduction in procedures lasts, and how its benefits and adverse events compare with existing care. The FDA’s accelerated approval status means those follow-up data remain an important part of the evidence record.
Key Questions
What is Voceryq approved to do?
The FDA approved Voceryq to reduce the frequency of surgical papilloma excisions in adults with recurrent respiratory papillomatosis. It is not described as a cure.
What did the trial find?
In the open-label phase I/II trial, 7 of 26 patients (27%) who had at least three surgeries in the preceding year had no surgical excisions during 12 months of follow-up. The figure does not describe all 32 trial participants.
Does treatment eliminate the need for surgery?
Not necessarily. The reported outcome was achieved by a subgroup, and the treatment regimen starts with surgery to remove visible papillomas. The FDA approval is intended to reduce the frequency of later excisions, not establish that surgery is no longer needed.
What side effects were reported?
Common adverse events in trials included injection-site reactions, headache, fatigue, hypertension, nausea and dizziness. Label warnings also include muscle contractions, transient bleeding and vasovagal reactions. The source report does not provide event rates.
What evidence is still required?
The FDA requires postmarketing trials to verify and describe the therapy’s clinical benefit. The available report does not specify the studies’ timeline or design.
Source: medpagetoday.com
