Inovio Gets FDA Approval for Rolling BLA Submission for Rare Disease Asset INO-3107

Inovio Pharmaceuticals has received FDA approval to submit a rolling BLA for its rare disease asset, INO-3107, for Recurrent Respiratory Papillomatosis. The company projects completion of the submission in the next few months.

INOVIO Pharmaceuticals (NASDAQ: INO) has received approval from the U.S. Food and Drug Administration (FDA) to begin the rolling submission of its Biologics License Application (BLA) for INO-3107, an investigational DNA medicine designed to treat Recurrent Respiratory Papillomatosis (RRP) in adults. The company anticipates completing the submission in the coming months and aims to receive priority review, with the goal of FDA file acceptance by the end of 2025 [1].

RRP is a rare and debilitating condition caused by human papillomavirus (HPV) infection, characterized by the development of small, wart-like growths in the respiratory tract. These papillomas can lead to severe airway obstruction and respiratory complications, significantly impacting patients' quality of life. Surgery is the standard treatment, but papillomas often regrow, posing risks to the vocal cords and requiring multiple surgeries [2].

INO-3107 is designed to elicit an antigen-specific T cell response against HPV-6 and HPV-11 proteins, aiming to prevent or slow the growth of new papillomas. In a Phase 1/2 trial, 72% of patients saw a 50-to-100% reduction in surgeries after one year, and this number increased to 86% after two years. The FDA has granted INO-3107 both Orphan Drug and Breakthrough Therapy designations, allowing for accelerated approval [1].

The approval of INO-3107 marks a significant milestone in the treatment landscape for RRP, which has historically lacked effective therapeutic options. INOVIO's proprietary DNA medicines platform, utilizing its CELLECTRA delivery device, offers a novel approach to treating RRP by teaching the body to manufacture its own disease-fighting tools [1].

References:
[1] https://www.marketscreener.com/news/inovio-to-begin-rolling-submission-of-bla-for-ino-3107-for-the-treatment-of-rrp-in-adults-ce7c50d9db88f62c
[2] https://www.theglobeandmail.com/investing/markets/markets-news/GetNews/34316850/recurrent-respiratory-papillomatosis-treatment-landscape-fda-approves-papzimeos-first-therapy-in-history-delveinsights-perspective-on-market-impact-competitive-landscape-and-pipeline-therapies/