Pharmacology

“It represents the first time an in vivo HSC-directed gene insertion therapy has been evaluated in a patient," said CEO Jim Burns in a statement.

A phase 1b study of zelpultide alfa demonstrated a favorable safety profile and encouraging early reductions in BPD and ventilation duration, supporting advancement to a pivotal international Phase 2b/3 program.

The agency approved a monthly dosing option for select patients acorss all approved indications, including wAMD, DME, DR, and RVO.

“This label expansion helps close a long-standing gap in the treatment of pediatric patients with hereditary antithrombin deficiency,” said George M. Rodgers, III, MD, PhD.

The target action date for roflumilast cream 0.3% for plaque psoriasis in children 2 to 5 years is set for June 29, 2026.

Dupilumab maintained histologic and endoscopic improvements across all pediatric age groups with EoE, according to Joshua Wechsler, MD.

Thomas Wallach, MD, discusses phase 3 safety data of tenapanor in pediatric patients with IBS-C, presented at the 2025 NASPGHAN Annual Meeting.

FDA approves first irritable bowel syndrome with constipation (IBS-C) treatment, linaclotide (Linzess), for children aged 7 years and older based on pediatric and adult trial data.

If approved, this UC indication would add to pediatric indications of ustekinumab for psoriasis and active psoriatic arthritis.

Explore groundbreaking therapies for type 1 diabetes that aim to delay onset, preserve β-cell function, and restore insulin independence.

Tezepelumab received FDA approval for CRSwNP in patients 12 years and older, supported by strong phase 3 data.

Combination vaccines enhance childhood immunization by reducing the number of shots, improving adherence, and potentially increasing overall vaccination rates.

Ibrahim Gwarzo, MBBS, DrPH, MPH, explains his study that found timely opioid administration was associated with lower hospitalization rates for children with uncomplicated sickle cell pain.

Prompt opioid dosing in emergency care may reduce hospital stays for children with sickle cell disease pain, new research revealed.

Ji-Hyun Lee reports that short surgical exposure to sevoflurane did not affect IQ, behavior, or language outcomes in infants and toddlers.

Results suggest that a single, brief exposure to general anesthesia for surgery is unlikely to impair short-term neurodevelopment in young children.

FDA approves first generic iron sucrose injection for treating iron deficiency anemia in CKD patients 2 years and older.

Explore essential resources and toolkits for managing the ongoing stimulant medication shortage affecting ADHD treatment and patient care.

Manufacturer MannKind has submitted a supplemental Biologics Application to the FDA for its inhaled insulin Afrezza.

Findings may help guide clinicians and policymakers in balancing the risks and benefits of stimulant prescribing in adolescent populations, according to the study authors.

Get caught up with Contemporary Pediatrics! This list helps you navigate our top stories from the week, all in one place.

A look back at the FDA submissions and regulatory decisions in the pediatric health care space from June 2025.

Lebrikizumab demonstrated efficacy and safety in patients with skin of color and moderate to severe atopic dermatitis in the ADmirable trial. Trial investigator Andrew Alexis, MD, MPH, reacts.

The approved indication now includes children down to 6 weeks of age to protect against invasive meningococcal disease via serogroups A, C, W, and Y.