A new oral formulation of Jorveza (budesonide, Dr. Falk Pharma GmbH) has received regulatory approval in the European Union for pediatric patients with eosinophilic esophagitis (EoE), marking a significant advancement in managing this rare inflammatory condition in this population.

At its February meeting, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending approval of Jorveza 0.2 mg/mL oral suspension for the treatment of EoE in children and adolescents aged 2-17 years.

The existing orodispersible tablet formulation (0.5-mg and 1-mg strengths) of Jorveza remains approved only for adults (≥ 18 years).

Addressing Pediatric Treatment Gaps

EoE is a chronic, immune-mediated esophageal disease characterized by eosinophil‑predominant inflammation, causing dysphagia, chest pain, food impaction, and upper abdominal discomfort. The condition has risen in prevalence over the past three decades and carries substantial healthcare costs. 

Until now, pediatric treatment options have been limited. To treat younger patients with EoE, clinicians often relied on off-label adult treatments, utilizing formulations outside their approved indications, dosages, or administration routes. 

Jorveza oral suspension is the first pediatric-specific, licensed formulation for EoE in the EU and is intended to reduce reliance on off‑label adult treatments. 

Jorveza contains budesonide, which is a corticosteroid that binds to immune cell receptors, reducing the release of inflammatory mediators. Administered as an oral suspension, the medication is designed to coat the esophageal mucosa via saliva, thereby reducing local inflammation and alleviating EoE symptoms.

Proven Histologic and Symptom Response

Clinical evidence supporting pediatric use comes from the phase 2 PEER study, which evaluated 71 patients aged 2-18 years over a 12-week period. The trial compared a placebo against low, medium, and high doses of the oral budesonide suspension.

Patients in the medium-dose (52.6%) and high-dose (47.1%) groups achieved significantly higher response rates than did placebo recipients (5.6%). Response was defined as a peak eosinophil count below 6 per high-power field and a more than 50% reduction in symptom scores.

Histologic improvement reached more than 50% in the medium-dose group and 90% in the high-dose group.

Safety Profile and Next Regulatory Steps

The suspension demonstrated a favorable safety profile with no unexpected concerns. Oral candidiasis was reported in approximately 3.3% of participants who received the suspension, a rate consistent with topical corticosteroid use. While small blood pressure increases were observed, they were clinically insignificant with no related adverse events. Notably, monitoring of growth velocity showed no differences in height changes between treatment groups during the study period.

Treatment should be initiated by clinicians experienced in diagnosing and managing EoE.

The medicine had an accelerated assessment, reflecting the need for development incentives. In addition, it received orphan designation, which indicated the rarity and seriousness of EoE. 

The European Commission is expected to issue a final marketing authorization decision within 67 days of the CHMP opinion. 

Detailed prescribing information in all EU languages will be provided in the updated Summary of Product Characteristics after approval by the commission.