Weekly review: New delgocitinib data for eczema, FDA action items, more | Image Credit: © Artur - © Artur - stock.adobe.com.

Thank you for visiting the Contemporary Pediatrics® website. Take a look at some of our top stories from last week (Monday, February 3, to Friday, February 7, 2025), and click on each link to read and watch anything you may have missed.

Phase 3: Delgocitinib improves chronic hand eczema in adolescents

Phase 3 trial results for delgocitinib (Anzupgo) 20 mg/g cream demonstrated significant efficacy in adolescents (ages 12–17) with chronic hand eczema (CHE), meeting primary and secondary endpoints in the DELTA TEEN study. The topical pan-JAK inhibitor, already approved for adults in several regions, showed promising results in improving moderate to severe CHE, a condition with considerable impact on quality of life. LEO Pharma aims to address the unmet need in this underserved patient group with further scientific publication and regulatory submissions.

Click here for the full article and additional data.

FDA grants ABO-101 orphan, rare pediatric designations to treat primary hyperoxaluria type 1

The FDA has granted orphan drug and rare pediatric disease designations to ABO-101, a gene-editing therapy for primary hyperoxaluria type 1 (PH1), a rare genetic kidney disease affecting children. Arbor Biotechnologies plans to initiate a Phase 1/2 trial in early 2025 to assess ABO-101’s safety, tolerability, and efficacy. The therapy, which utilizes CRISPR-Cas12i2 technology to reduce oxalate production, aims to provide a durable treatment option for this life-threatening condition.

Click here for the full report.

Positive data reported for Trappsol Cyclo in patients 3 years and under with NPC1

Preliminary phase 3 data from Cyclo Therapeutics' sub-study on Trappsol Cyclo suggest potential benefits for children under three with Niemann-Pick Disease Type C1 (NPC1). The study found stabilization or improvement in most patients, particularly those with mild to severe disease, supporting early intervention to slow disease progression. If interim results from the broader TransportNPC trial meet statistical significance, the company plans to pursue FDA and EMA approval.

FDA approves tocilizumab-anoh as biosimilar for multiple diseases

The FDA has approved tocilizumab-anoh (Avtozma; Celltrion) as a biosimilar to Actemra, with indications for rheumatoid arthritis, juvenile idiopathic arthritis, giant cell arteritis, and severe COVID-19. Phase 3 trial data confirmed its efficacy, safety, and pharmacokinetic comparability to the reference drug, supporting its approval in both intravenous and subcutaneous forms. While offering expanded treatment options, the biosimilar carries a risk of serious infections, particularly in immunosuppressed patients.

Click here for more approval details.

Experts: Now is the time to be the biggest child health care advocate

Pediatric health experts stress the importance of scientific integrity, proactive advocacy, and vaccinations in the face of misinformation and shifting public perceptions. Leaders in the field emphasize engaging parents with accurate information, advocating for public health, and reinforcing the critical role of vaccines in preventing severe childhood diseases. Despite challenges, health care professionals remain committed to evidence-based medicine and safeguarding children’s well-being.

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