Long-term Safety and Efficacy of Budesonide Oral Suspension for Eosinophilic Esophagitis: A 4-Year, Phase 3, Open-Label Study.

BACKGROUND & AIMS: We investigated the long-term safety and efficacy of budesonide oral suspension (BOS) in eosinophilic esophagitis (EoE).

METHODS

This study (SHP621-303) was a 4-year, phase 3, open-label study in patients with EoE who completed up to 52 weeks of BOS therapy in 2 preceding phase 3 studies. On the basis of treatment assignments in previous studies, patients were assigned to BOS-BOS or placebo-BOS groups. All patients received BOS 2.0 mg twice daily; dose reductions to once daily and interruptions were permitted. The safety and tolerability of BOS were primarily investigated, with exploratory efficacy endpoints also examined.

RESULTS

Overall, 131 patients were included. BOS was well-tolerated, with no unexpected safety signals observed. Treatment-emergent adverse events (TEAEs) occurred in 76.3% of patients; most were mild/moderate in severity and unrelated to study drug. The most frequently reported BOS-related TEAEs included abnormal adrenocorticotropic hormone stimulation test results (8.4%, 11/131; number of events [m] = 12) and adrenal insufficiency (2.3%, 3/131; m = 3). Esophageal candidiasis occurred in 3.1% of patients (4/131). The aforementioned TEAEs resolved in most patients. At month 48 of treatment, 50.0% and 58.3% of patients achieved/maintained a histologic response (≤6 and <15 eosinophils per high-power field, respectively). The initial reduction (-3.6) in total EoE Endoscopic Reference Score from baseline to the first visit was maintained until month 48.

CONCLUSIONS

Long-term treatment with BOS was well-tolerated. Despite dosing changes/interruptions, approximately half of patients achieved/maintained a histologic response; initial improvements in endoscopic outcomes were maintained over 48 months.

CLINICALTRIALS: gov number: NCT03245840.