A recap of the top clinical trial and pipeline data readouts affecting pediatric care during September 2026.
News|Videos|July 28, 2025
WATCH: Experts react to the FDA approval of sepiapterin to treat PKU
Fact checked by: Joshua Fitch, Senior Editor
Suzanne Hollander, MS, RD, LDN; and Timothy Tramontana, MD, offer thoughts on newly FDA-approved sepiapterin for PKU.
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Upadacitinib showed continued facial and body repigmentation in NSV through week 76, with numerically higher responses when combined with NB-UVB.
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.
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