INO-3107 Clears FDA's BLA Review Hurdle: What This Means for RRP Patients

INOVIO Pharmaceuticals has achieved a significant regulatory milestone with the FDA’s acceptance of the Biologics License Application for INO-3107, its investigational immunotherapy targeting recurrent respiratory papillomatosis. The acceptance letter, delivered recently, sets an October 30, 2026 PDUFA date and clears the pathway for expedited review under accelerated approval status—though the regulatory agency flagged that supplementary data may be required to fully justify this accelerated designation.

Understanding the Clinical Challenge

Recurrent respiratory papillomatosis represents a serious unmet medical need. This rare condition, predominantly triggered by HPV-6 and HPV-11 viral strains, causes relentless growth of benign tumors throughout the respiratory tract. These growths obstruct airways and impair voice quality, severely diminishing patient quality of life. Currently, repeated surgical interventions remain the only standard treatment option. However, surgery carries substantial risks—tissue damage to vocal cords accumulates with each procedure, and the disease frequently recurs, trapping patients in a cycle of operations.

Clinical Evidence Supporting the BLA Submission

The regulatory submission rests on robust Phase 1/2 trial data. Among adults with RRP who had undergone minimum two surgeries within the preceding year, the therapy demonstrated compelling results: 72% of participants achieved between 50-100% reduction in surgical interventions during the initial year post-treatment. More impressively, longer-term follow-up analysis revealed that 86% of evaluable patients sustained clinical improvements into the second year without requiring repeat doses—and notably, half of these patients needed zero additional surgeries.

Research findings appeared in Nature Communications and The Laryngoscope, providing peer-reviewed validation of INO-3107’s potential impact.

Mechanism and Safety Profile

INO-3107 functions as an immunotherapy designed to mobilize antigen-specific T-cell responses directly targeting HPV-6 and HPV-11-infected cells. By activating this cellular immunity, the treatment aims to suppress or halt papilloma proliferation at its source. Clinical tolerability data proved encouraging, with predominantly mild adverse events including localized injection-site reactions and fatigue—no serious safety signals emerged.

Regulatory Status and Financial Position

The FDA granted INO-3107 dual designations reflecting its potential significance: Orphan Drug status and Breakthrough Therapy classification. INOVIO’s balance sheet showed $50.8 million in combined cash, equivalents, and short-term investments as of September 30, 2025, projected to sustain operations through Q2 2026.

Despite the BLA acceptance, market participants reacted cautiously. Stock performance reflected concern about the FDA’s caveat regarding additional information requirements for accelerated approval eligibility. INO shares have ranged between $1.30-$2.98 over the past 12 months and currently trade in pre-market activity at $1.85, representing a 19.21% decline.

Next Steps

INOVIO intends to convene with FDA officials to clarify remaining information needs and accelerate the path toward potential approval. The company expressed confidence that INO-3107 addresses a genuine therapeutic gap for this rare respiratory disease population.