By Alex Philippidis - February 26, 2025 - The FDA has granted Priority Review to Precigen’s Biologics License Application (BLA) for its lead candidate PRGN-2012, the company said—a step forward for a gene therapy that, if approved, would be the first treatment indicated for adults with the rare disease of recurrent respiratory papillomatosis (RRP).
The FDA also set an August 27 target date for deciding on Precigen’s BLA under the Prescription Drug User Fee Act (PDUFA). PRGN-2012—which Precigen has begun to call by its generic name of zopapogene imadenovec—is a gene therapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11.
Infection with HPV 6 or HPV 11 causes RRP, a lifelong neoplastic disease of the upper and lower respiratory tracts that can be fatal. According to Precigen, some 27,000 U.S. adults have RRP based on a recently updated internal analysis derived from claims data, and more than 125,000 patients outside the United States have the disease. The standard of care for RRP patients consists of numerous surgeries.
Precigen has projected a “multi-billion-dollar global blockbuster potential” for PRGN-2012. The publicly traded company has not guided investors to a projected list price for the gene therapy, though president and CEO Halen Sabzevari, PhD, told GEN Edge recently that the company has focused on late clinical development and eventual commercialization of PRGN-2012 since the summer.
In July, Precigen announced the appointment of Phil Tennant as chief commercial officer, with initial duties focused on overseeing commercial readiness activities for the potential launch of PRGN-2012.
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