Precigen, a biopharmaceutical company specializing in the development of innovative gene and cell therapies, has announced that the European Commission (EC) has granted Orphan Drug Designation for the company’s first-in-class investigational medicine PRGN-2012 for the treatment of recurrent respiratory papillomatosis (RRP).
RRP is a rare, difficult-to-treat and sometimes fatal neoplastic disease of the upper and lower respiratory tracts caused by human papillomavirus type 6 (HPV 6) or HPV type 11 (HPV 11). PRGN-2012 received Orphan Drug Designation from the United States Food and Drug Administration (FDA) and Precigen was the first company to receive Breakthrough Therapy Designation and an accelerated approval pathway from the FDA for an RRP treatment.
PRGN-2012 is an innovative therapeutic vaccine with optimized antigen design that uses gorilla adenovector technology, part of Precigen’s proprietary AdenoVerse™ platform, to elicit immune responses directed against cells infected with HPV 6 or HPV 11. Gorilla adenovectors have numerous advantages, including the ability for repeat administration, the inability to replicate in vivo, and the ability to deliver a large genetic payload.
Orphan Drug Designation in the European Union (EU) is granted by the EC based on a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products. Medicines intended for the treatment, diagnosis or prevention of seriously debilitating or life-threatening conditions that affect fewer than five in 10,000 people in the European Union (EU) are eligible for the designation. The Orphan Drug Designation allows companies certain regulatory, financial and commercial incentives to develop medicines for rare diseases where there are no satisfactory treatment options.
“Outside of the US, our commissioned research suggests that there are more than 125,000 patients diagnosed and living with RRP, a rare and debilitating disease which currently has no approved therapeutic treatment option and is treated through repeated surgical intervention,” said Helen Sabzevari, PhD, President and CEO of Precigen.
“The ongoing Phase 1/2 study for PRGN-2012 in the United States has shown the immense potential for this therapy. Results from the Phase 1 study showed significant clinical benefit with 50% of patients remaining surgery-free after more than two years following treatment.
“Additionally, PRGN-2012’s favorable safety profile and ease of administration are potentially paradigm shifting for both patients and their treating physicians. This designation is an important next step as we work to expand this novel therapeutic to the EU in hopes of benefiting the unmet needs of RRP patients.”
PRGN-2012 is currently under investigation in a Phase 1/2 pivotal single-arm study in adult patients with RRP in the United States. A Phase 2 data presentation is anticipated in the second quarter of 2024 and a planned Biologics License Application (BLA) submission under an accelerated approval pathway with the FDA is anticipated in the second half of 2024. Commercial readiness preparations are underway for a potential launch in the United States in 2025.