Precigen secures full USFDA nod for Papzimeos for adults with Recurrent Respiratory Papillomatosis

RRP is a rare, debilitating, and potentially life-threatening disease of the upper and lower respiratory tract caused by chronic HPV 6 or HPV 11 infection. RRP can lead to severe voice disturbance, a compromised airway, and recurrent post-obstructive pneumonias. Management of RRP has primarily consisted of repeated surgeries, which do not address the root cause of the disease and can be associated with significant morbidity as well as significant patient and health system burden.

"For more than a century, since RRP was first recognized as a distinct disease, patients have had to rely on repeated surgeries to manage this relentless condition. Today marks a historic turning point. With the landmark FDA approval of PAPZIMEOS and broad label, all adult RRP patients are now eligible for access to the first and only approved therapy that targets the root cause of the disease," said Helen Sabzevari, PhD, President and CEO of Precigen. "This milestone affirms the power of our AdenoVerse platform and the exceptional capabilities of our team to rapidly advance a wholly novel therapy from discovery to approval considerably faster than industry benchmarks. We are profoundly grateful to the NIH clinicians, the FDA, and—most importantly—the patients and families who made this breakthrough possible. We look forward to swiftly delivering PAPZIMEOS to the RRP community and ushering in a new era of treatment that targets the underlying cause of the disease rather than just managing its symptoms."

"This long-awaited FDA approval represents a momentous milestone for the RRP community," said Kim McClellan, President of the Recurrent Respiratory Papillomatosis Foundation. "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. We are deeply grateful to the teams at Precigen and the NIH, and above all, to the patients and caregivers whose courage, advocacy, and perseverance have made this historic moment possible."

The approval is supported by data from the open-label, single-arm, pivotal study in adult patients with RRP:

• The pivotal study successfully met its primary safety and pre-specified primary efficacy endpoints.
• 51% (18 out of 35) of study patients achieved Complete Response, requiring no surgeries in the 12 months after treatment with PAPZIMEOS. These Complete Responses remained durable for over 12 months. Of the 18 patients with a Complete Response in the ongoing study, 15 patients evaluated at 24 months demonstrated continued Complete Response.
• PAPZIMEOS was well-tolerated with no dose-limiting toxicities and no treatment-related adverse events greater than Grade 2.
• PAPZIMEOS induced HPV 6/11-specific T cell responses in RRP study patients with a significantly greater expansion of peripheral HPV-specific T cells in responders compared with non-responders.

The pivotal study was led by lead investigators, Clint T. Allen, MD, and Scott M. Norberg, DO, at the National Institutes of Health. Pivotal data were presented at the 2024 American Society of Clinical Oncology (ASCO) annual meeting and published in The Lancet Respiratory Medicine.