August 22nd 2025
In recent studies, donidalorsen (DAWNZERA) has demonstrated durable efficacy and long-term disease control.
August 21st 2025
High-dose centanafadine significantly improved ADHD symptoms in children aged 6–12 and was well tolerated, according to a phase 3 clinical trial.
August 19th 2025
If approved, vatiquinone would have been the first therapy approved for pediatric patients with FA.
August 14th 2025
Richard Auchus, MD, PhD, discusses how crinecerfont is improving CAH care and easing the transition from pediatric to adult management.
August 12th 2025
FDA approves first generic iron sucrose injection for treating iron deficiency anemia in CKD patients 2 years and older.
FDA approves brensocatib to treat adolescents with non–cystic fibrosis bronchiectasis
Brensocatib was granted FDA priority review in February 2025 for the small molecule, oral, reversible inhibitor.
FDA accepts priority review for gepotidacin as oral treatment for gonorrhea
FDA accepts priority review of gepotidacin, a first-in-class oral antibiotic, for treating uncomplicated urogenital gonorrhea in patients ≥12 years.
FDA approves tocilizumab-anoh for CRS in patients 2 years and older
The expanded label of tocilizumab-anoh now includes treatment for patients aged 2 years and older with cytokine release syndrome (CRS).
FDA approves fremanezumab-vfrm for migraine prevention in children aged 6 years, older
The indication approved is for children aged 6 years and older who weigh at least 45 kg (99 lbs) for the preventive treatment of episodic migraine.
Media and mental health, hemophilia approval, July FDA recap, and more
Get caught up with Contemporary Pediatrics! This list helps you navigate our top stories from the week, all in one place.
FDA news in pediatrics: July 2025
A look back at the FDA submissions and regulatory decisions in the pediatric health care space from July 2025.
FDA approves concizumab-mtci for hemophilia A/B without inhibitors in patients 12 years and older
The FDA approved concizumab-mtci (Alhemo; Novo Nordisk) for hemophilia A/B without inhibitors, offering once-daily subcutaneous prophylaxis that significantly reduces bleeding rates.
FDA grants fast track designation to rezpegaldesleukin for severe alopecia areata in patients 12 years or older
Nektar Therapeutics remains on track to announce top-line phase 2 data in December.
FDA approves pegcetacoplan as first treatment for C3 glomerulopathy or IC-MPGN
Pegcetacoplan (Empaveli; Apellis Pharmaceuticals) is indicated for patients 12 years or older to reduce proteinuria.
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
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