August 1st 2025
A look back at the FDA submissions and regulatory decisions in the pediatric health care space from July 2025.
July 31st 2025
The FDA approved concizumab-mtci (Alhemo; Novo Nordisk) for hemophilia A/B without inhibitors, offering once-daily subcutaneous prophylaxis that significantly reduces bleeding rates.
July 29th 2025
Nektar Therapeutics remains on track to announce top-line phase 2 data in December.
Pegcetacoplan (Empaveli; Apellis Pharmaceuticals) is indicated for patients 12 years or older to reduce proteinuria.
Suzanne Hollander, MS, RD, LDN; and Timothy Tramontana, MD, offer thoughts on newly FDA-approved sepiapterin for PKU.
FDA approves sepiapterin to treat phenylketonuria in adult, pediatric patients
“Without treatment, individuals with PKU would have very high blood Phe levels...so that results in neurocognitive deficits and mood disorders," Suzanne Hollander, MS, RD, LDN, said.
FDA approves avatrombopag for pediatric immune thrombocytopenia, including new sprinkle formulation
The FDA has approved avatrombopag and new sprinkle formulation for children ≥1 year with chronic ITP, offering an oral, food-flexible treatment option.
FDA grants fast track designation to SGT-501 gene therapy for CPVT
Solid Biosciences is planning a phase 1b clinical trial of SGT-501 to treat the catecholaminergic polymorphic ventricular tachycardia heart condition.
FDA approves gardenia blue as natural color additive for food use
The FDA approves gardenia (genipin) blue as a natural food color, enhancing options for manufacturers and supporting the shift away from synthetic dyes.
FDA-approved berdazimer gel, 10.3% launches to treat molluscum contagiosum
The nitric oxide–releasing topical is the first prescription medication approved for at-home treatment of molluscum contagiosum.
FDA accepts NDA for ET-600, desmopressin oral solution for pediatric AVP deficiency
The FDA accepted Eton Pharmaceuticals' NDA for ET-600 for pediatric arginine vasopressin deficiency, with a target action date in February of 2026.
FDA approves sebetralstat as first and only on-demand treatment for HAE
Though the original target action date was not met by the FDA, KalVista's sebetralstat is now approved to treat HAE in patients 12 years and older.
Key FDA approvals in pediatric health: First half of 2025
A roundup of the most impactful FDA approvals in pediatric health from January through June 2025, spanning diagnostics, therapeutics, and at-home care.
FDA news in pediatrics: June 2025
A look back at the FDA submissions and regulatory decisions in the pediatric health care space from June 2025.
MNV-201 receives fast track, rare pediatric disease to treat Pearson Syndrome
MNV-201 is being evaluated in phase 2 clinical trials for treatment of the ultra-rare, life-threatening mitochondrial disorder affecting children.
Mercedes Gonzalez, MD, discusses phase 2 trial of roflumilast cream 0.05% for infant atopic dermatitis
Roflumilast cream 0.05% is being studied for mild-to-moderate AD in infants aged 3 months to under 2 years in a new phase 2 trial.
FDA approves at-home belimumab autoinjector for children with lupus nephritis
The FDA has approved belimumab (Benlysta; GSK) autoinjector for children with lupus nephritis, expanding at-home treatment.
Weekly review: New PrEP for HIV, new HAE treatment, and more
Get caught up with Contemporary Pediatrics! This list helps you navigate our top stories from the week, all in one place.
FDA extends review of ruxolitinib cream for pediatric atopic dermatitis
The FDA has extended the review of ruxolitinib for atopic dermatitis in children 2 to 11 years to September 2025 to evaluate new CMC data.
FDA approves twice-yearly lenacapavir as PrEP for HIV in adolescents, adults
Lenacapavir is the first twice-yearly option available in the US for those who need or want PrEP, according to Gilead Sciences.
Phase 3 trial of omaveloxolone to treat Friedreich ataxia in children aged 2 to <16 begins
Currently, omaveloxolone (Skyclarys) is approved to treat adults and adolescents aged 16 years and older impacted by FA.
FDA accepts NDA for epinephrine sublingual film to treat type 1 allergic reactions
The federal agency has assigned a target action date of January 31, 2026, for a decision.
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.