FDA news in pediatrics: July 2025 | Image Credit: © Contemporary Pediatrics.

Another month has come and gone, and with it, several FDA approvals and regulatory updates related to the ever-evolving landscape of pediatric health care.

In our July monthly recap, we list our top FDA-related news items in a quick, easy-to-read format so you can stay informed.

Take a look at our detailed coverage of FDA-related news from July and easily stay in touch with our digital newsletters that bring you practical information for today's pediatrician.

Click the title of each story below for our full coverage of that regulatory update.

FDA updates in pediatric care: July 2025

1. FDA revises labeling on extended-release stimulants for ADHD to include weight loss risk

The FDA will update labeling for all extended-release attention-deficit/hyperactivity disorder (ADHD) stimulants to include warnings about weight loss risk in children under 6 years, the agency announced in July 2025.

Though not approved for this age group, these medications are often prescribed off-label. FDA findings showed higher plasma exposure and increased adverse events, including significant weight loss, in young children taking extended-release amphetamine and methylphenidate. As a result, new limitations of use will be added or revised to reflect these risks, and clinicians are advised to monitor growth and consider alternative treatments when managing ADHD in this population.

2. FDA approves sebetralstat as first and only on-demand treatment for HAE

On July 7, 2025, the FDA approved sebetralstat (Ekterly; KalVista Pharmaceuticals) as the first oral, on-demand treatment for hereditary angioedema (HAE) attacks in patients 12 years and older.

Previously, HAE treatments required injection. Approval was based on the phase 3 KONFIDENT trial, which showed faster symptom relief and attack resolution with sebetralstat compared with placebo. The therapy was well tolerated and demonstrated a similar safety profile to placebo. The approval follows a delayed review and marks a significant advance in patient-controlled HAE management.

3. FDA accepts NDA for ET-600, desmopressin oral solution for pediatric AVP deficiency

On July 8, 2025, the FDA accepted Eton Pharmaceuticals’ new drug application for ET-600, an oral liquid formulation of desmopressin for treating central diabetes insipidus (AVP-D) in pediatric patients.

The NDA has a target action date of February 25, 2026. If approved, ET-600 would be the first FDA-approved oral liquid desmopressin for children, addressing dosing challenges associated with current therapies and meeting a significant unmet need in pediatric endocrinology.

4. FDA approves gardenia blue as natural color additive for food use

On July 14, 2025, the FDA approved gardenia (genipin) blue as a color additive in food, the fourth natural-source dye authorized in recent months.

The approval allows use in beverages, candies, and teas, supporting efforts to phase out synthetic, petroleum-based dyes. Derived from the gardenia fruit, gardenia blue was approved under section 721 of the FD&C Act following a petition by the Gardenia Blue Interest Group. The decision aligns with HHS’s broader initiative to prioritize natural additives and public health.

5. J&J submits icotrokinra NDA for plaque psoriasis in patients 12 years or older

On July 21, 2025, Johnson & Johnson submitted a new drug application to the FDA for icotrokinra, an investigational oral peptide targeting the IL-23 receptor, for moderate to severe plaque psoriasis in patients 12 years and older.

The submission is supported by positive results from four phase 3 trials in the ICONIC program, which showed significant skin clearance and a favorable safety profile. If approved, icotrokinra would be the first oral IL-23–targeting therapy for psoriasis, offering a new, once-daily option for patients seeking effective, noninjectable treatment.

6. FDA grants fast track designation to SGT-501 gene therapy for CPVT

On July 23, 2025, the FDA granted fast track designation to Solid Biosciences’ investigational gene therapy SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT), a rare and potentially fatal inherited arrhythmia.

SGT-501 uses an AAV-based vector to deliver a functional CASQ2 gene to heart muscle cells, aiming to stabilize calcium regulation disrupted in CPVT. The therapy also holds orphan drug and rare pediatric disease designations. With no FDA-approved treatments currently available for CPVT, SGT-501 may offer a first-in-class precision approach targeting the underlying genetic cause.

7. FDA approves avatrombopag for pediatric immune thrombocytopenia, including new sprinkle formulation

On July 28, 2025, the FDA approved avatrombopag (Doptelet) for treating thrombocytopenia in pediatric patients 1 year and older with persistent or chronic immune thrombocytopenia (ITP) unresponsive to prior therapy.

The approval includes a new oral granule formulation, Doptelet Sprinkle, for children aged 1 to under 6 years. Supported by phase 3 trial data, Doptelet demonstrated significant platelet response and was well tolerated. This marks the first pediatric approval for the oral thrombopoietin receptor agonist, expanding treatment options with flexible, age-appropriate formulations.

8. FDA approves sepiapterin to treat phenylketonuria in adult, pediatric patients

On July 28, 2025, the FDA approved sepiapterin (Sephience; PTC Therapeutics) to treat phenylketonuria (PKU) in both adult and pediatric patients, including infants as young as 1 month with sepiapterin-responsive hyperphenylalaninemia (HPA).

The approval, supported by phase 3 APHENITY trial data, includes broad labeling across PKU subtypes. Sepiapterin, used alongside a phenylalanine-restricted diet, significantly increased natural protein intake while maintaining blood phenylalanine control, offering a new therapeutic option that may help reduce reliance on medical formula and improve quality of life for patients across all ages.

9. FDA approves pegcetacoplan as first treatment for C3 glomerulopathy or IC-MPGN

On July 28, 2025, the FDA approved pegcetacoplan (Empaveli; Apellis Pharmaceuticals) as the first treatment for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years and older.

The approval, based on results from the phase 3 VALIANT study, showed a 68% reduction in proteinuria and stabilization of kidney function. Pegcetacoplan also demonstrated substantial clearance of C3 deposits. This marks a significant advance for patients with these rare kidney diseases, who previously had no FDA-approved treatment options.