FDA Accepts BLA for Precigen’s Recurrent Respiratory Papillomatosis Gene Therapy PRGN-2012 With Priority Review
The PDUFA action date for the BLA has been set at August 27, 2025.
The FDA has accepted the biologics license application (BLA) that Precigen submitted for PRGN-2012, a gene therapy product intended to treat recurrent respiratory papillomatosis (RRP) with priority review.1 The Prescription Drug User Fee Act (PDUFA) action date for the BLA has been set at August 27, 2025, and no advisory committee meeting is currently planned for the BLA.
PRGN-2012 is based on the company’s AdenoVerse platform and is intended to trigger an immune response targeted at cells infected by human papillomavirus (HPV) 6 or HPV 11, thus providing an alternative to the current standard of care for RRP, which typically involves repeated surgical interventions. The BLA for PRGN-2012 is supported by results from a phase 1/2 clinical trial (NCT04724980). The study, which reached its primary completion date on June 5, 2024, enrolled 38 patients in total. It remains active, but is no longer recruiting new patients.
"The priority review designation is a testament to the FDA's recognition of the significant unmet need for the RRP patient population,” Helen Sabzevari, PhD, the president and chief executive officer of Precigen, said in a statement.1 “RRP patients have never had an FDA-approved therapy, relying instead on repeated surgeries to alleviate the symptoms of RRP without addressing the underlying disease. Treatment with PRGN-2012 has shown significant, durable clinical benefit. We have patients treated with PRGN-2012 who have been surgery-free for more than 3 years now, bringing hope for an alternative to the cycle of repeated surgeries, which carry immense risk for irreversible damage and significant morbidity. We look forward to working with the FDA over the coming months during their BLA review and hope to introduce the first FDA-approved therapeutic option to the RRP patient population, estimated at more than 27,000 adults in the United States, later this year."
Data from the phase 1/2 trial were previously presented by Precigen at the 2024 American Society of Clinical Oncology (ASCO) annual meeting at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting, held May 31 to June 4, in Chicago, Illinois. Precigen reported that over 50% of patients treated in the trial attained a complete response (CR) and over 85% of patients had a decrease in surgical interventions in the year after treatment with the gene therapy, when contrasted with the year before receiving the gene therapy. The CR rate (CRR) was defined as the percentage of patients who did not require any RRP surgery during the 12 months after receiving the gene therapy. With regard to safety, PRGN-2012 was characterized as “well-tolerated”. There were no dose-limiting toxicities and no treatment-related adverse events assessed as grade 3 or higher in severity. The CRR and safety constituted the study’s primary end points, and both of these were met.
"We are thrilled with the results of the phase 1/2 pivotal study showing more than half of patients were surgery free–CR–and 86% of patients had a significant reduction in the need for surgeries after PRGN-2012 treatment,” Sabzevari said in a June 2024 statement.2 “Based on the efficacy, safety, and ease of administration, we believe PRGN-2012 is a game-changer for RRP patients and has the potential to be the preferred treatment-of-choice for RRP. We look forward to sharing these results with the FDA as part of a rolling BLA submission under an accelerated approval pathway. We have ramped up our commercial readiness efforts in anticipation of a potential launch in 2025 and are excited by the potential to bring a long overdue new treatment option to the RRP community."
In August 2024, Precigen announced that it was putting its focus on advancing PRGN-2012 through potential commercialization.3 As a result, the company paused several of its UltraCAR-T clinical programs and all preclinical programs. Furthermore, the company indicated it was letting go of more than 20% of its employees.
"We are on track toward our goal of submitting a rolling BLA for PRGN-2012 in the second half of this year and we are pleased to announce that the confirmatory clinical trial, an important step guided by the FDA to support an accelerated approval, has already been initiated and is actively enrolling patients," Sabzevari in a statement at the time.3 "These prioritization steps enhance our ability to rapidly prepare for potential commercialization of PRGN-2012, which if approved, we believe has the safety, efficacy, and route of administration profile to be the first and best-in-class therapy for RRP patients."
