September 8th 2025
Get caught up with Contemporary Pediatrics! This list helps you navigate our top stories from the week, all in one place.
September 6th 2025
The FDA approves recombinant VWF for pediatric von Willebrand disease, enhancing treatment options for children during bleeding episodes and surgeries.
September 3rd 2025
Arcutis has submitted an sNDA to the FDA for roflumilast cream 0.3% for plaque psoriasis in children aged 2 to 5 years.
September 1st 2025
A look back at the FDA submissions and regulatory decisions in the pediatric health care space from August 2025.
August 22nd 2025
In recent studies, donidalorsen (Dawnzera) has demonstrated durable efficacy and long-term disease control.
FDA approves garadacimab-gxii to prevent HAE attacks in patients aged 12 years, older
Garadacimab-gxii is the only treatment to target factor Xlla for prophylactic use to prevent attacks of HAE in patients aged 12 years and older.
FDA will not meet original PDUFA date for sebetralstat to treat HAE
Though the decision will not meet the June 17, 2025, target date, the federal agency anticipates a decision within approximately 4 weeks.
FDA approves new presentation of ustekinumab-stba biosimilar for plaque psoriasis, psoriatic arthritis
The approval for ustekinumab-stba now offers all dosage forms to the reference product, ustekinumab, and is indicated for patients aged 6 to 17 years.
Weekly review: Clesrovimab for RSV approved, new dupilumab data, and more
Bavarian Nordic initiates trial of chikungunya vaccine in children aged 2-11 years
Currently, Vimkunya is FDA-approved for use among individuals aged 12 years and older.
FDA expands glecaprevir/pibrentasvir indication for acute hepatitis C down to 3 years of age
Glecaprevir/pibrentasvir was a "highly efficacious treatment for people with acute HCV," with 96.2% of patients analyzed having sustained virological response 12 weeks post-treatment.
First infant enrolled in trial of roflumilast cream, 0.05% for atopic dermatitis in infants
The phase 2 trial will be 4 weeks and evaluate investigational, once-daily roflumilast cream 0.05% in infants 3 months to less than 2 years of age.
FDA approves clesrovimab to protect infants during first RSV season
The BLA for clesrovimab was based on results demonstrated in the phase 2b/3 CLEVER trial among healthy pre-term and full-term infants.
Dupilumab improves atopic dermatitis severity in adolescents, adults with skin of color
Results at 24 weeks revealed that 76% of individuals achieved a 75% or more improvement in overall disease severity (EASI-75), trial's primary endpoint.
Positive data: Navepegritide, lonapegsomatropin combination treatment for children with achondroplasia
The combination therapy yielded boosted treatment benefits of navepegritide, including improvement in mean ACH Z-score and mean annualized growth.
Six-month IXCHIQ chikungunya vaccine data demonstrates sustained antibody levels
Six-month results from the phase 2 VLA1553-221 trial aligned with initial data and demonstrated immune response at day 180 among patients aged 1 to 11 years.
FDA approves pediatric indication for SONU wearable device to treat nasal congestion
The AI-enabled, wearable device is the first FDA-approved, drug-free solution for nasal congestion and allergies in children ages 12 and up.
FDA accepts navepegritide application to treat children with achondroplasia
With the new drug application accepted with priority review, the FDA has assigned a date of November 20, 2025, to complete its review for potential approval.
FDA expands Xenoview indication down to 6 years for MRI lung ventilation evaluation
The hyperpolarized contrast agent for oral inhalation can now be used in patients 6 years and older, lowering the approved indication from 12 years of age.
FDA approves hydrocortisone oral solution for children 5 years, older with adrenocortical insufficiency
The decision makes hydrocortisone oral solution (Khindivi; Eton Pharmaceuticals) the only FDA-approved oral formulation of hydrocortisone.
Reaction: FDA approves roflumilast foam 0.3% for scalp, body psoriasis
Review some of the reactions from experts about the recent FDA approval of roflumilast foam 0.3% to treat scalp and body psoriasis in patients aged 12 years or older.
FDA approves MenQuadfi meningococcal vaccine for children 6 weeks or older
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.
Weeky review: COVID-19 vaccine approved, framework laid out, and more
FDA approves roflumilast foam 0.3% for scalp, body psoriasis
The once-daily, steroid-free topical is now approved to treat plaque psoriasis of the scalp and body in patients aged 12 years and older.
FDA approves Jivi for patients aged 7 years and older with hemophilia A
The expanded indication follows original approval in August 2018 for use in previously-treated patients 12 years and older.
Weekly review: Multiple FDA action items, USPSTF recommendation, and more
FDA approves NVX-CoV2705 COVID-19 vaccine
NVX-CoV2705 is indicated for individuals aged 12 to 64 years with an underlying condition that poses high risk for severe COVID-19 outcomes.
Duchenne muscular dystrophy gene therapy demonstrates improved motor functions
Delandistrogene moxeparvovec-rokl (ELEVIDYS) showed significant motor function improvements in 8- to 9-year-old Duchenne muscular dystrophy patients in part 2 of the phase 3 EMBARK study.
Phase 3 data show effectiveness of somapacitan in children with growth disorders
Somapacitan was non-inferior and had a similar safety profile and clinical outcomes compared to once-daily somatropin in improving yearly growth rates.
INZ-701 shows early promise in treating ENPP1 Deficiency in pediatric patients
Safety, phosphate level gains, and immunogenicity data offer encouraging signals in the pivotal trial.
FDA accepts NDA for berotralstat in HAE patients aged 2 to 11 years
The federal agency provided BioCryst Pharmaceuticals a target action date of September 12, 2025 for potential approval of berotralstat (ORLADEYO).
FDA approves pediatric indication for Optison ultrasound enhancing agent
This FDA decision will help to improve clarity and diagnostic accuracy of echocardiograms in pediatric patients, according to GE HealthCare.
FDA clears EN-374 therapy application for X-linked chronic granulomatous disease
The in vivo hematopoietic stem cell-directed therapy received rare pediatric disease and orphan drug designations earlier this year.
New data support crinecerfont for pediatric congenital adrenal hyperplasia
Subgroup data show crinecerfont helps children with CAH reduce steroid use while maintaining or improving androstenedione levels across diverse patient groups.
INF108 to prevent necrotizing enterocolitis receives FDA designations
NEC is a life-threatening condition marked by the death of intestinal tissue, which impacts preterm infants.