Long-term efficacy and safety of omalizumab for nasal polyposis in an open-label extension study

ABSTRACT Background Chronic rhinosinusitis with nasal polyps (CRSwNP) frequently remains uncontrolled despite maximal medical therapy and sinonasal surgery, presenting several unmet needs and challenges. Omalizumab previously demonstrated efficacy in CRSwNP in duplicate, phase 3, randomized, placebo-controlled trials (POLYP 1, POLYP 2). Objective This open-label extension evaluated continued efficacy, safety, and durability of response of omalizumab in adults with CRSwNP who completed POLYP 1 or 2. Methods After 24 weeks of omalizumab or placebo in POLYP 1 and 2, patients (n = 249) received open-label omalizumab plus background nasal mometasone for 28 weeks and were subsequently followed for 24 weeks after omalizumab discontinuation. Efficacy endpoints assessed change from baseline for the coprimary endpoints, Nasal Polyp Score and Nasal Congestion Score, and the secondary endpoints of Sino-Nasal Outcome Test-22, Total Nasal Symptom Score and its components, and University of Pennsylvania Smell Identification Test scores. Safety objectives included incidence of adverse events (AEs) and AEs leading to omalizumab discontinuation. Results Patients who continued omalizumab experienced further improvements across coprimary endpoints and secondary endpoints through 52 weeks. Patients who switched from placebo to omalizumab experienced favorable responses across endpoints through week 52 that were similar to POLYP 1 and 2 at week 24. After omalizumab discontinuation, scores gradually worsened over the 24-week follow-up, but remained improved from pretreatment levels for both groups. The safety profile was similar to previous reports. Conclusion The efficacy and safety profile from this study supports extended omalizumab treatment up to 1 year for CRSwNP with inadequate response to nasal corticosteroids.