Precigen Submits Priority Application to FDA for PRGN-2012: A Breakthrough in RRP Treatment

Precigen Makes Strides Towards FDA Approval of PRGN-2012 for RRP

Precigen, Inc., a leading biopharmaceutical company, has taken a significant step in the fight against recurrent respiratory papillomatosis (RRP) by submitting a biologics license application (BLA) to the US Food and Drug Administration (FDA) for its investigational therapy, PRGN-2012. This submission, made on December 30, 2024, marks the potential emergence of the first approved therapeutic option for adult patients suffering from this challenging and burdensome disease.

What is Recurrent Respiratory Papillomatosis?

RRP is a rare chronic disorder characterized by the growth of benign tumors in the respiratory tract, primarily caused by infections with human papillomavirus (HPV) types 6 and 11. These tumors can lead to severe respiratory complications and often require numerous surgical interventions to manage. Unfortunately, the existing standard-of-care—repeated surgeries—does not tackle the root cause, necessitating a cycle of procedures that can occur hundreds of times over a patient's lifetime. This adds a significant burden not only to the patients but also to their caregivers and families.

PRGN-2012: A New Hope

PRGN-2012, also known by its international nonproprietary name zopapogene imadenovec, is an innovative gene therapy employing the AdenoVerse® platform. This investigational treatment aims to stimulate the immune system to target cells infected with HPV. With both Breakthrough Therapy Designation and Orphan Drug Designation from the FDA, PRGN-2012 has a unique regulatory pathway that indicates its potential benefits over existing therapies.

The BLA submission includes a request for priority review, which, if approved, would shorten the review period from ten to six months, expediting access for patients. This process is critical as there are currently no FDA-approved treatments for RRP, and patients are left to endure frequent surgeries with limited options for relief from this debilitating condition.

Supporting Evidence from Clinical Trials

The data underpinning the BLA comes from a pivotal Phase 1/2 clinical trial involving adult patients with RRP. In this study, more than half of the participants experienced a complete response, meaning they no longer required surgeries within a year following PRGN-2012 treatment. Additionally, more than 85% of patients required fewer surgical interventions compared to the year before treatment. The results showcased that PRGN-2012 was well-tolerated, with no serious red flags regarding adverse effects.

Looking Ahead

Helen Sabzevari, PhD, President and CEO of Precigen, emphasized the importance of the BLA submission, noting that the impact of RRP on patients and families has been neglected for decades. The company's recent financial maneuvers have also positioned it well for potential commercialization, with hopes to launch PRGN-2012 by the second half of 2025.

As Precigen awaits the FDA's response, the company is optimistic about the future of PRGN-2012, envisioning it as a game-changer for those grappling with RRP. This submission not only represents a step forward in therapy for RRP but also showcases the promise of precision medicine in addressing complex and underserved medical needs in society. With advancements like PRGN-2012, the future of RRP treatment looks brighter, giving hope to patients and families who have long awaited effective solutions.

For more information about Precigen and its innovations, visit Precigen's website or their profiles on social media platforms.