Upadacitinib showed continued facial and body repigmentation in NSV through week 76, with numerically higher responses when combined with NB-UVB.
News|Articles|October 1, 2026
Clinical trial news in pediatrics: September 2026
Author(s)Morgan Ebert, Executive Editor
Fact checked by: Benjamin P. Saylor
A recap of the top clinical trial and pipeline data readouts affecting pediatric care during September 2026.
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September 2026's pediatric clinical trial readouts clustered heavily around growth and metabolic disorders. Three skeletal dysplasia programs — two in achondroplasia and one in hypochondroplasia — reported growth-velocity gains, while two GLP-1-class agents posted substantial BMI reductions in adolescents and in children as young as 6. Rare pediatric epilepsy, kidney disease, and eosinophilic esophagitis trials each delivered positive secondary or post hoc results, and a post-exposure COVID-19 antiviral's supporting phase 3 data rounded out the month.
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A recap of the top FDA approvals/regulatory decisions/pipeline updates affecting pediatric care during September 2026.
Joseph Rossano, MD, discusses how FDA approval of mavacamten introduces a disease-targeted treatment option for adults and pediatric patients weighing 30 kg (66 lbs) or more with symptomatic oHCM.
FDA approves Camzyos for pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy
Mavacamten becomes the first cardiac myosin inhibitor approved for adults and pediatric patients weighing 30 kg (66 lbs) or more with symptomatic oHCM.
In a 48-patient phase 1b trial, ecnoglutide was well tolerated in adolescents with obesity and reduced BMI up to 12.6% at 20 weeks.
DBV filed a BLA for the Viaskin peanut patch in peanut-allergic children aged 4-7, citing phase 3 VITESSE data, and requested priority review.
ScolimiR-3D, a 6-microRNA blood test to predict severe curve progression in AIS, received FDA Breakthrough Device designation.
The FDA approved tiratricol (Emcitate) for peripheral thyrotoxicosis in MCT8 deficiency, the first approved therapy for the rare disorder.
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