#USA #Germantown #RRP #Precigen #PRGN-2012

FDA Grants Priority Review to Precigen's BLA for PRGN-2012

In a significant advancement for the treatment of recurrent respiratory papillomatosis (RRP), the U.S. Food and Drug Administration (FDA) has granted priority review to Precigen, Inc.'s biologics license application (BLA) for its investigational gene therapy, PRGN-2012 (also known as zopapogene imadenovec). This designation is a remarkable accomplishment as it reduces the review timeline to just six months, indicating an urgency to evaluate therapies that could provide substantial improvements to the healthcare landscape, particularly for serious and chronic conditions. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of August 27, 2025.

RRP is an infrequent but debilitating disease characterized by the growth of wart-like lesions in the respiratory tract, which are caused by human papillomaviruses (HPV) types 6 and 11. Those afflicted with RRP often face a lifetime of recurring surgeries to manage their condition, with no current FDA-approved therapies available to mitigate symptoms or tackle the root cause of the disease. The current treatment paradigm, which involves repeated surgical procedures, can lead to significant morbidity and potential laryngeal injury, particularly after multiple surgeries over the years.

The data supporting the BLA for PRGN-2012 emerges from a pivotal Phase 1/2 clinical trial, detailed at the American Society of Clinical Oncology (ASCO) annual meeting, and subsequently published in The Lancet Respiratory Medicine. The results of this study are promising, with over 50% of patients achieving a complete response—defined as no need for further surgeries within the year following PRGN-2012 treatment. Furthermore, a staggering 85% of study participants reported a reduction in surgical interventions the year subsequent to their treatment.

Notably, PRGN-2012 has not only received priority review from the FDA, but it has also secured Breakthrough Therapy Designation and Orphan Drug Designations, which are a testament to the potential benefits of this therapy. Implicit in the high unmet need for effective treatment options is the acknowledgment that RRP patients could drastically benefit from a pharmacological approach that addresses their condition non-surgically. As Helen Sabzevari, PhD, President and CEO of Precigen, articulated, the designation of priority review underscores the urgent necessity for an FDA-approved therapy tailored for RRP patients who have historically relied on surgery.

Patient anecdotes reflect the pressing need: some individuals have endured countless surgeries throughout their lives, living in a constant cycle of recurrence and intervention. However, with PRGN-2012, there is renewed hope, as some patients treated have not required surgery for over three years following their initial treatment with this innovative therapy.

The AdenoVerse® platform from Precigen facilitates the effective delivery of this gene therapy via proprietary adenovectors. These vectors have shown superior performance in generating robust and durable immune responses, leveraging UltraVector® technology, which may offer a sustainable therapeutic solution for RRP. Importantly, this could alleviate not only the burden of frequent surgeries but also the associated risks and complications that come with them.

As Precigen prepares for the upcoming review process, anticipation builds within the healthcare community and the RRP patient population—estimated to include over 27,000 adults in the U.S. alone. Their optimism is tinged with the potential for a drastic shift in their treatment experience, paving the path for a future where surgeries are not the only option.

With the FDA's current stance indicating they do not intend to convene an advisory committee meeting for discussions regarding this application, the focus pivots to the ongoing review, with stakeholders keenly observing the outcomes. Precigen’s mission to offer innovative, precision-based therapies to treat pressing health issues underscores their commitment to advancing medicine, distinctly exemplified in their pursuit of gaining FDA approval for PRGN-2012.

In conclusion, the forthcoming months will be pivotal not only for Precigen but also for the thousands of adults grappling with RRP. There lies hope that the review of PRGN-2012 will culminate in the FDA's green light for this much-needed treatment, heralding a new era of therapeutic options for patients burdened by this critical illness.