Two significant clinical advances are reshaping treatment options for patients with rare inflammatory conditions. A phase 2 clinical trial published in The Lancet Gastroenterology & Hepatology found that dupilumab, a monoclonal antibody already approved for other conditions, significantly improved outcomes for people with eosinophilic gastritis (EoG), a rare food-allergic inflammatory disease of the stomach. Meanwhile, the FDA approved roflumilast cream for plaque psoriasis in children as young as age 2, marking the first steroid-free topical option for this age group.

What is eosinophilic gastritis and why is treatment so difficult?

Eosinophilic gastritis is a rare condition in which the stomach becomes inflamed due to an allergic immune response, causing a surge in eosinophils, a type of white blood cell. For people with EoG, common foods like dairy, wheat, eggs, soy, fish, and nuts can trigger severe reactions including nausea, vomiting, abdominal pain, and fatigue. Until now, patients have had no FDA-approved medications to address the underlying inflammation. Instead, they have relied on strict, lifelong food-elimination diets and symptom management, which can lead to poor growth, anemia, and nutritional deficiencies over time.

The rarity of EoG makes drug development challenging. An estimated 5 to 7 people per 100,000 in the population have the condition, making it difficult for pharmaceutical companies to justify the cost of large clinical trials needed for FDA approval. This is why researchers turned to studying an already-approved drug in a new disease setting.

How did dupilumab perform in the eosinophilic gastritis trial?

The Dupilumab Eosinophilic Gastritis Study (DEGAS) involved 41 teenagers and adults with EoG treated at 11 medical centers across the United States. Researchers randomly assigned participants to receive either dupilumab or a placebo for 12 weeks, after which all participants received dupilumab during an open-label extension phase lasting through 36 weeks.

Those who received dupilumab instead of placebo during the initial 12-week period showed measurable improvements in objective clinical markers. The key findings included:

• Stomach eosinophil counts: Participants showed reduced numbers of eosinophils in stomach tissue samples, indicating the drug was addressing the underlying inflammatory process.
• Endoscopy results: Visual examination of the stomach showed improved findings in those receiving active treatment compared to placebo.
• Tissue pathology: Microscopic examination of stomach tissue samples revealed improved scores in the dupilumab group, with these improvements continuing through 36 weeks of treatment.
• Safety profile: The medication was well-tolerated, with a safety profile consistent with previous studies of dupilumab in other conditions.

Dupilumab works by blocking signaling from interleukin-4 and interleukin-13, key cytokines involved in type 2 inflammation. The drug was first approved for atopic dermatitis (eczema) in 2017 and has since been approved for eight additional conditions, including eosinophilic esophagitis (EoE), a related condition affecting the food pipe.

"For many rare diseases, the small numbers of people affected makes it extremely difficult for pharmaceutical companies to conduct the clinical studies needed to secure U.S. FDA approval. But for some conditions, investigational evaluation of an already-approved therapy in a new, relevant disease setting can become a vital pathway to improved outcomes," said Marc Rothenberg, MD, PhD, director of the Division of Allergy and Immunology at Cincinnati Children's.

Marc Rothenberg, MD, PhD, Director of the Division of Allergy and Immunology at Cincinnati Children's

The study was conducted by the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR), a collaboration within the NIH-supported Rare Diseases Clinical Research Network. This collaborative approach allowed researchers to conduct rigorous trials more efficiently by sharing research infrastructure across 11 participating centers.

What does the roflumilast approval mean for young children with psoriasis?

On June 29, 2026, the FDA approved roflumilast cream 0.3% (ZORYVE) for plaque psoriasis in children as young as age 2, closing a significant prescribing gap for pediatric dermatologists. Until now, clinicians treating psoriasis in very young children faced an uncomfortable choice: topical steroids that work but carry well-documented risks when applied to thin or sensitive skin areas, or systemic medications poorly suited to toddlers.

What makes this approval clinically meaningful is not just the age extension but the absence of site-specific restrictions. Topical steroids are not recommended for long-term use on the face, skin folds, and genitals, precisely the areas where psoriasis in young children tends to concentrate. Roflumilast cream carries no such limitations, making it the only topical phosphodiesterase-4 (PDE4) inhibitor indicated for plaque psoriasis in children under 6, with explicit approval for use on intertriginous areas, or skin folds.

The approval rests on safety and efficacy data from multiple studies. The FDA reviewed a 4-week Maximal Usage Systemic Exposure study (ARQ-151-216) in children ages 2 to 5 with plaque psoriasis covering at least 2% of body surface area, supplemented by 24 weeks of open-label safety data from an extension study. The FDA also referenced efficacy data from the DERMIS-1 and DERMIS-2 Phase 3 trials, which evaluated the cream in patients aged 2 and older using an Investigator's Global Assessment (IGA) success measure, defined as clear or almost clear skin with at least a 2-grade improvement from baseline.

How to monitor roflumilast use in young children

While the approval expands treatment options significantly, experts recommend tracking real-world data as the medication enters routine clinical practice:

• Systemic exposure monitoring: The MUSE study was designed to confirm systemic exposure stays within acceptable bounds under maximal-use conditions in controlled settings, not to generate long-term pharmacokinetic surveillance in everyday use.
• Post-marketing surveillance: If post-marketing data in routine care shows meaningful systemic accumulation in toddlers, particularly when application may be less controlled than in a study setting, the current label's unrestricted-duration language may face pressure to change.
• Clinical follow-up: Dermatologists should continue monitoring patients for any unexpected systemic effects as the drug is used more widely in the youngest age groups.

Arcutis, the company behind roflumilast, has now secured two distinct pediatric roflumilast franchises in overlapping patient populations. The company previously received approval for a 0.05% roflumilast cream for atopic dermatitis in children ages 2 to 5, meaning clinicians now have a steroid-free topical option for multiple inflammatory skin conditions in this age group.

What comes next for both treatments?

For dupilumab and eosinophilic gastritis, the research team notes that while tissue-level improvements are encouraging, larger trials will be needed to better assess symptom relief and long-term effects. The findings support continued study of targeted, immune-based approaches and raise important questions about optimal dosing, treatment duration, and how tissue-level improvements translate to patient experience.

"While this study focused on changes seen in stomach tissue, the results provide important groundwork for larger trials that can better assess symptoms and long-term effects," explained Nirmala Gonsalves, MD, an esophageal expert and Professor of Medicine at Northwestern University School of Medicine.

Nirmala Gonsalves, MD, Professor of Medicine at Northwestern University School of Medicine

Both advances illustrate how clinical research networks and collaborative approaches can accelerate progress for rare diseases. By bringing together investigators with specialized expertise and sharing research infrastructure, researchers can conduct rigorous trials more efficiently, potentially serving as a model for addressing the 7,000-plus rare diseases that still lack effective treatments.