FDA Approves Budesonide Oral Suspension (Eohilia) for Eosinophilic Esophagitis

Ikuo Hirano, MD

Credit: Northwestern Medicine

The US Food and Drug Administration (FDA) has approved budesonide oral suspension (Eohilia) for patients ≥ 11 years of age with eosinophilic esophagitis (EoE), making it the first and only FDA-approved oral therapy for this patient population.¹

According to the February 12 release from Takeda Pharmaceuticals, significantly more patients receiving budesonide oral suspension achieved histologic remission and a greater absolute change from baseline in patient-reported Dysphagia Symptom Questionnaire (DSQ) combined score after 12 weeks of treatment compared to those receiving placebo.¹

“Various formulations of corticosteroids have been used in the past to manage EoE, but in an off-label capacity and using multiple delivery options. With EOHILIA, it’s gratifying to now have an FDA-approved treatment specifically formulated for a consistent dose delivery with demonstrated ability to address esophageal inflammation and EoE dysphagia symptoms,” said Ikuo Hirano, MD, professor of medicine and director of the Kenneth C Griffin Esophageal Center in the Division of Gastroenterology and Hepatology at Northwestern University Feinberg School of Medicine.¹ “As the treatment needs and goals of patients with EoE can vary, I welcome the flexibility that EOHILIA offers as an oral medication.”

Of note, the FDA approved dupilumab (Dupixent) as the first and only approved medicine specifically indicated for pediatric EoE patients 1-11 years of age weighing ≥ 15 kg on January 25. This decision expanded upon the fully human monoclonal antibody’s prior approval in adult and pediatric patients 12 years and older weighing ≥ 40 kg in May of 2022, when it became the first and only medicine specifically indicated to treat EoE in the United States.^2,3

The approval is based on efficacy and safety data from a pair of multicenter, randomized, double-blind, parallel-group, placebo-controlled 12-week studies in patients ages 11-56 years and 11-42 years, respectively, with EoE, labeled Study 1 and Study 2. In both studies, patients received ≥ 1 dose of either budesonide oral suspension 2 mg twice daily or placebo orally twice daily.¹

Efficacy endpoints included histologic remission, defined as peak eosinophil count of ≤6 per high-powered field across all available esophageal levels, and the absolute change from baseline in patient-reported DSQ combined score after 12 weeks of treatment. The DSQ measures how often a patient with EoE has trouble swallowing and the behavioral adaptations they subsequently use, as reported directly by patients.¹

Results showed significantly more patients receiving budesonide oral suspension achieved histologic remission vs placebo in both Study 1 (53.1% vs 1%) and Study 2 (38% vs 2.4%). Additionally, absolute change from baseline in DSQ combined score in the budesonide oral suspension groups was greater than in the placebo groups (Study 1, -10.2 vs -6.5, respectively, and Study 2, -14.5 vs -5.9, respectively). During the last 2 weeks of each study, more patients receiving budesonide oral suspension experienced no dysphagia or only experienced dysphagia that “got better or cleared up on its own” as compared to placebo, as measured by the DSQ.¹

As noted in the release, budesonide oral suspension has not been shown to be safe and effective for the treatment of EoE for longer than 12 weeks. The most common adverse reactions in Study 1 included respiratory tract infection (13%), gastrointestinal mucosal candidiasis (8%), headache (5%), gastroenteritis (3%), throat irritation (3%), adrenal suppression (2%) and erosive esophagitis (2%). The safety profile of budesonide oral suspension in Study 2 was generally similar to that observed in Study 1.¹

“For most of us, eating is a simple experience. But for people living with eosinophilic esophagitis, sitting down for a meal can include painful and difficult swallowing, chest pain and a choking sensation,” said Brandon Monk, senior vice president and head of the US Gastroenterology Business Unit at Takeda.¹ “With EOHILIA, patients and their physicians now have the first and only FDA-approved oral treatment option for EoE that was shown during two 12-week clinical studies to reduce esophageal inflammation and improve the ability to swallow.”

It was additionally noted in the release that budesonide oral suspension will be available in 2 mg/10 mL single-dose stick packs by the end of February.¹

References:

1. ^{Takeda Pharmaceuticals. FDA Approves Takeda’s EOHILIA (budesonide oral suspension), the First and Only Oral Treatment in the U.S. for Eosinophilic Esophagitis (EoE). Newsroom. February 12, 2024. Accessed February 12, 2024. https://www.takeda.com/newsroom/newsreleases/2024/fda-approves-eohilia/}
2. ^{Fitch, J. FDA approves dupilumab for eosinophilic esophagitis in children aged 1 to 11 years. Contemporary Pediatrics. January 25, 2024. Accessed February 12, 2024. https://www.contemporarypediatrics.com/view/fda-approves-dupilumab-for-eosinophilic-esophagitis-in-children-aged-1-to-11-years}
3. ^{Brooks, A. Dupilumab's Role in Managing Pediatric EoE, with Evan Dellon, MD, MPH. HCPLive. January 25, 2024. Accessed February 12, 2024. https://www.hcplive.com/view/dupilumab-s-role-in-managing-pediatric-eoe-with-evan-dellon-md-mph}