Morgan Ebert, Executive Editor

BioMarin reports 3-year vosoritide growth data in hypochondroplasia and early BMN 333 findings in achondroplasia.

pCPA gains FDA Orphan Drug Designation for monoamine oxidase deficiency, signaling early development for an ultrarare neurodevelopmental disorder.

Diazoxide choline showed sustained hyperphagia improvements in Prader-Willi syndrome after randomized withdrawal and retreatment.

Setmelanotide reduced BMI measures, fat mass, and hyperphagia scores in interim phase 2 Prader-Willi syndrome data.

A feasibility study found acoustic resonance therapy improved nasal congestion and rhinitis symptoms in adolescents without adverse events.

The FDA cited Happiest Baby, Inc. for distributing unevaluated X-Small and X-Large sleep sacks, marketing its bassinet for hospital use without authorization, and multiple quality system violations including reports of mold and unsanitary refurbished units.

The FDA has approved gadoquatrane (Ambelvist), a next-generation macrocyclic GBCA that delivers effective CNS and whole-body MRI contrast at 60% less gadolinium than standard macrocyclic agents.

Physicians treating infants who consumed Nara Organics Whole Milk Organic Powdered Infant Formula should initiate treatment based on clinical presentation and not await laboratory confirmation.

The FDA granted accelerated approval to teplizumab (Tzield) for children aged 8–17 with recently diagnosed stage 3 T1D, based on PROTECT phase 3 data.

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

A CHOP-led study identified IL-6 and IFN-γ immune activation tied to worse recovery in children with sepsis or organ failure.

A first-in-human study of 96 preterm infants shows broadband optical spectroscopy can safely distinguish NEC from healthy tissue within 2 minutes at bedside, without pain or radiation.

New ADA 2026 analyses show Afrezza delivered comparable glycemic control, favorable safety, and higher treatment satisfaction in youth.

The FDA has approved expanded indications for tocilizumab-bavi (TOFIDENCE), adding severe or life-threatening CAR T-cell–induced CRS and hospitalized COVID-19 in patients aged 2 years and older.

The FDA has accepted Takeda's supplemental application for IV vedolizumab in patients ages 2 and older with moderate-to-severe UC or Crohn's disease, with a PDUFA date set for Q1 2027.

The FDA has approved an every-8-week maintenance regimen for lebrikizumab-lbkz (EBGLYSS), giving eligible adults and adolescents with moderate-to-severe atopic dermatitis as few as 6 injections per year without mandatory topical therapy.

The FDA has approved bemotrizinol as the first new active sunscreen ingredient under the OTC monograph in nearly two decades, clearing the broad-spectrum UV filter for use in adults and children 6 months of age and older.

FDA designations for investigational GEn-1123 may support development in Duchenne muscular dystrophy, but clinical data remain limited.

New research using a natural experiment design found consistent reductions in diagnosed influenza among vaccinated young children in every season studied from 2016 to 2023.

Vertex reported phase 3 ALYFTREK data in children aged 2 to 5 years with cystic fibrosis and responsive CFTR genotypes.

A cross-sectional study comparing 2 Australian population-based cohorts found that updated infant feeding guidelines recommending egg introduction by 6 months were associated with a 17.7% relative reduction in egg allergy prevalence.

The FDA has broadened the approved indication for marstacimab (Hympavzi; Pfizer) to include pediatric patients ages 6–11 with hemophilia A or B and patients 12 and older with inhibitors.

A look back at the latest clinical trial updates for the pediatric population during May 2026.

Common pediatric skin infections are often managed in primary care, but some presentations require dermatology referral and further evaluation.

Take a quick look at everything you may have missed in May, including the top FDA approvals and latest clinical updates.

FDA approved ensitrelvir for COVID-19 post-exposure prophylaxis in patients aged 12 years and older after positive phase 3 trial results.

A look back at the FDA submissions and regulatory decisions in the pediatric health care space from May 2026.

The FDA accepted an sNDA for mavacamten in adolescents with obstructive hypertrophic cardiomyopathy, with a decision expected in September 2026.

This decision marks the first FDA-approved inhaled insulin for pediatric patients.

The FDA extended its review of adrabetadex for infantile-onset Niemann-Pick disease type C to November 17, 2026, after a major NDA amendment.