FDA approves budesonide oral suspension for eosinophilic esophagitis

For the second time in 3 weeks, the FDA has approved a treatment for eosinophilic esophagitis (EoE) in pediatric patients.¹

Takeda announced that the FDA has approved its budesonide oral suspension (EOHILIA) as the first and only oral therapy approved by the federal agency for adults and pediatric patients aged 11 years and older with EoE.

The twice-daily novel formulation is a corticosteroid indicated for 12 weeks of treatment. The formulation of budesonide confers thixotropic properties allowing more flow when shaken, returning "to a more viscous state when swallowed," the company stated in a news release.

Beyond 12 weeks, budesonide oral suspension "has not been shown to be safe and effective for the treatment of EoE," Takeda stated in the press release.

Approval is based on a pair of randomized, multicenter, double-blind, parallel-group, and placebo-controlled studies that lasted 12 weeks. Study 1 took place in patients aged 11 to 56, while Study 2 in those aged 11 to 42 with EoE.

Each study featured the receipt of at least 1 dose of either budesonide oral suspension 2 mg twice daily or placebo orally twice daily.

Peak eosinophil count of 6 or less high-powered field across all available esophageal levels (histologic remission) and the absolute change from baseline in patient-reported Dysphagia Symptom Questionnaire (DSQ) combined score after 12 weeks of treatment were efficacy outcomes.

In Study 1, 53.1% of patients who received budesonide oral suspension achieved histologic remission compared to 1% of patients who recevied placebo.

Thirty-eight percent of patients that received budesonide oral suspension in Study 2 achieved histologic remissions compared to 2.4% of patients in the placebo group.

In study 1, the absolute change from baseline in DSQ combined score in the budesonide oral suspension group was –10.2 (1.5) compared to –6.5 (1.8) in the placebo group.

In study 2, absolute change was from baseline in DSQ was –14.2 (1.8) compared to –5.9 (2.1) in the budesonide oral suspension and placebo groups, respectively.

"With [budesonide oral suspension], 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, Kenneth C. Griffin Esophageal Center, Division of Gastroenterology and Hepatology, Northwestern University Feinberg School of Medicine in the news release.

“As the treatment needs and goals of patients with EoE can vary, I welcome the flexibility that [budesonide oral suspension] offers as an oral medication,” said Hirano.

In ≥2% of patients receiving budesonide oral suspension and at a rate greater than placebo, the most common adverse reactions included respiratory tract infection (13%), gastrointestinal mucosal candidiasis (8%), headache (5%), gastroenteritis (3%), throat irritation (3%), adrenal suppression (2%), and erosive esophagitis (2%).

According to Takeda, budesonide oral suspension is cotraindicated in patients with hypersensitivity to budesonide. The company noted serious hypersensitivity reactions, including anaphylaxis, have occured with oral budesonide products.

The company stated, "Erosive esophagitis occurred in subjects who received EOHILIA in a 12-week clinical trial. None of the subjects had erosions at baseline esophagogastroduodenoscopy (EGD), and most were receiving concomitant therapy with a proton pump inhibitor (PPI)."

On Januaruy 25, 2024, the FDA approved dupilumab (Dupixent; Regeneron and Sanofi) for the treatment of EoE in children aged 1 to 11 years weighing at least 35 lbs (15 kg), which generated excitement from experts.²

References:

1. FDA approves Takeda's EOHILIA (budesonide oral suspension), the first and only oral treatment in the US for eosinophilic esophagitis (EoE). Takeda. Press release. 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