The Food and Drug Administration (FDA) has granted Orphan Drug designation to INO-3107 (Inovio), a DNA plasmid immunotherapy, for the treatment of recurrent respiratory papillomatosis, a rare disease caused by human papillomavirus (HPV) types 6 and 11 infections.

Recurrent respiratory papillomatosis (RRP) is characterized by small, noncancerous papillomas in the respiratory tract that lead to life-threatening airway obstructions. Recent findings from a pilot clinical study showed that INO-3106, a DNA medicine candidate targeting HPV 6-associated RRP, generated immunogenicity and engagement and expansion of an HPV 6-specific cellular response, including cytotoxic T cells. Treatment with the immunotherapy allowed 1 patient to delay surgery for over 1.5 years and another to remain surgery-free for over 2.5 years, according to a paper published in the journal Vaccines. Before the intervention, both of these patients needed to have surgery to remove tumor growths approximately every 180 days.

An open-label, multicenter phase 1/2 trial is currently open to enrollment and will evaluate the efficacy, safety, tolerability, and immunogenicity of INO-3107 in adult patients with HPV 6 and/or 11-associated RRP who have required at least 2 surgical interventions per year for the past 3 years for the removal of associated papilloma(s). The study expects to enroll 63 patients in the US. 

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Ami Shah Brown, PhD, MPH, INOVIO’s Senior Vice President, Regulatory Affairs, said, “Receiving FDA’s Orphan Drug designation for INO-3107 is an important milestone in the development of Inovio’s DNA medicine for this rare disease and clearly underscores the importance of addressing the unmet medical need for this debilitating condition.”

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1. Inovio receives Orphan Drug designation from US FDA for DNA medicine INO-3107 to treat rare disease recurrent respiratory papillomatosis (RRP). Accessed July 31, 2020.