[ad_1]
August 15, 2025
2 min read
The FDA approved the adenoviral vector-based immunotherapy, zopapogene imadenovec-drba, for treatment of certain patients with recurrent respiratory papillomatosis.
The indication applies to adult patients, making the agent the first and only FDA-approved therapy for the treatment of adults with recurrent respiratory papillomatosis (RRP). Zopapogene imadenovec-drba (Papzimeos, Precigen) is a first-of-its-kind non-replicating adenoviral vector-based immunotherapy, according to its manufacturer.
The FDA approved the adenoviral-based immunotherapy zopapogene imadenovec-drba for treatment of certain patients with recurrent respiratory papillomatosis.
RRP is a “rare, chronic disease caused by persistent human papillomavirus (HPV) 6 or 11 infection, leading to the growth of benign tumors in the respiratory tract, most commonly the larynx,” noted the FDA press release.
“This approval has the potential to transform the treatment landscape for RRP and offer lasting relief for patients who previously faced repeated surgeries to control symptoms of their disease,” Vinay Prasad, MD, MPH, director of the FDA’s Center for Biologics Evaluation and Research, said in a press release.
The agency based its approval on results from a single-arm, open-label trial which evaluated the agent in adults with RRP who required three or more surgeries each year.
“Randomized trials are not always needed to approve medical products and this approval is proof of that philosophy,” Prasad noted in the release. “The FDA will always demand the correct clinical study for the specific medical product and disease. Our requirements for products given to tens of millions of healthy people will be different than products given to at most hundreds or thousands of patients with unique diseases.”
Patients received four subcutaneous injections of zopapogene imadenovec-drba over a 12-week period following surgical debulking procedures.
During the pivotal portion of the study, data demonstrated that zopapogene imadenovec-drba met its primary safety and pre-specified primary efficacy endpoints.
Among the 35 patients who received the agent, 18 (51.4%) achieved a complete response and required no further surgery in the 12 months following treatment. Among the 18 patients who achieved a complete response, 15 patients evaluated at 24 months demonstrated a continued complete response.
Researchers confirmed the agent has a “favorable” safety profile, with most treatment-emergent adverse events being mild to moderate. No dose-limiting toxicities were observed, and no treatment-related serious adverse events were reported.
The approval was completed under priority review and the agent received both orphan drug and breakthrough therapy designations.
“This long-awaited FDA approval represents a momentous milestone for the RRP community,” Kim McClellan, president of the Recurrent Respiratory Papillomatosis Foundation, said in a manufacturer release. “For the first time, adult patients with RRP have access to an FDA-approved therapy that offers the potential to reduce — or even eliminate — endless repeated surgeries. This breakthrough brings long-overdue hope to patients and families who have endured so much.”
References:
[ad_2]
Source link
