As we close out 2025, Q4 brought several treatment regulatory updates and clinical trial results to help better take care of pediatric patients across the globe.

In the vast realm of pediatric health care, several specialty areas, from dermatology to allergy and immunology to infectious disease, saw newly FDA-approved medicines and updated clinical data for treatments still in development.

In the Q4 recap below, Contemporary Pediatrics highlights some of the top FDA-related action items, clinical trial data, and expert interviews conducted during the fourth quarter of 2025.

Click on each title below for additional information based on each news article.

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Q4 2025 regulatory updates in pediatrics

FDA approves Waskyra as first gene therapy for Wiskott-Aldrich syndrome

The FDA has approved Waskyra (etuvetidigene autotemcel; Fondazione Telethon), the first cell-based gene therapy authorized in the United States for Wiskott-Aldrich syndrome, for pediatric patients aged 6 months and older and eligible adults lacking a suitable matched donor. The approval was supported by open-label clinical studies and expanded access data showing substantial and durable clinical benefit, including marked reductions in severe infections and bleeding events following treatment with autologous, genetically corrected hematopoietic stem cells. The decision introduces a first-in-class therapeutic option for this rare, life-threatening immunodeficiency, where treatment had previously been limited to supportive care or donor-dependent stem cell transplantation.

FDA approves linaclotide (Linzess) for pediatric patients aged 7 years and older with IBS-C

The FDA has approved linaclotide (Linzess; Ironwood Pharmaceuticals) for the treatment of irritable bowel syndrome with constipation in pediatric patients aged 7 years and older, making it the first therapy authorized for IBS-C in this age group. The decision was supported by phase 3 trial data demonstrating significant improvements in spontaneous bowel movement frequency, stool consistency, and early treatment response compared with placebo, with a safety profile consistent with adult use. The approval expands treatment options for children and adolescents with IBS-C, a common and often challenging functional gastrointestinal disorder with limited prior pharmacologic therapies.

FDA approves roflumilast cream 0.05% for atopic dermatitis for children aged 2 to 5 years

The FDA has approved roflumilast cream 0.05% (Zoryve; Arcutis Biotherapeutics) for the treatment of atopic dermatitis in children aged 2 to 5 years, providing a topical, nonsteroidal option that can be used on any body area without duration limits. The decision was supported by phase 3 INTEGUMENT-PED and long-term extension data showing rapid improvement as early as week 1, significantly higher rates of clear or almost clear skin and EASI-75 response versus vehicle, and sustained efficacy through 56 weeks with a favorable safety profile. The approval expands treatment options for young children with widespread disease, for whom long-term topical steroid use may be inappropriate.

Q4 2025 top headlines in pediatrics

Review finds no systemic risks from aluminum in childhood vaccines

A recent review published in Pediatrics reaffirmed the safety and clinical value of aluminum adjuvants in childhood vaccines, concluding that nearly a century of evidence supports their role in enhancing immune responses without increasing the risk of toxicologic, neurologic, developmental, allergic, or autoimmune outcomes. Drawing on pharmacokinetic, epidemiologic, and clinical data, the authors found no association between aluminum-adjuvanted vaccines and autism spectrum disorder, asthma, allergic disease, or proposed autoimmune syndromes, noting that cumulative aluminum exposure from the pediatric immunization schedule remains low and is efficiently cleared by the body. The review emphasized that aluminum adjuvants are particularly important in early infancy to ensure durable protection and highlighted clear, empathetic clinician communication as central to maintaining caregiver trust and vaccine confidence.

ACIP votes to update hepatitis B vaccine recommendation for infants

The Advisory Committee on Immunization Practices voted to update its recommendation for hepatitis B vaccination in infants born to women who test negative for hepatitis B surface antigen, endorsing individualized, shared decision-making regarding the birth dose rather than universal administration. The December 5 vote drew criticism from pediatric and public health leaders, including the American Academy of Pediatrics, who cited insufficient evidence to support changing longstanding policy and warned the decision could undermine vaccine confidence. While ACIP approved revised guidance and related Vaccines for Children resolutions, the AAP reaffirmed its recommendation that all newborns receive the hepatitis B vaccine within 24 hours of birth, emphasizing decades of safety and effectiveness data.

FDA, CDC investigating multistate infant botulism outbreak tied to formula

The U.S. Food and Drug Administration and the Centers for Disease Control and Prevention have advised parents and caregivers to immediately stop using all ByHeart infant formula products amid an ongoing multistate outbreak of infant botulism. As of December 17, 2025, 51 infants across 19 states had been hospitalized with suspected or confirmed botulism linked to exposure to ByHeart Whole Nutrition Infant Formula, with epidemiologic and laboratory data suggesting possible contamination with Clostridium botulinum. Federal officials emphasized that infant botulism is a rare but life-threatening condition and urged caregivers to seek immediate medical attention if symptoms develop, while clinicians were advised to promptly report suspected cases for urgent consultation.