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 November 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 November 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: November 2025

1. J&J submits sBLA for ustekinumab to treat children with ulcerative colitis 2 years and up

On October 31, 2025, Johnson & Johnson submitted a supplemental Biologics License Application to the FDA to expand approval of ustekinumab (Stelara) for children aged 2 years and older with moderately to severely active ulcerative colitis (UC). The submission aims to address a major unmet need in pediatric inflammatory bowel disease, where nearly 20% of UC cases occur in children, who often present with more severe and aggressive disease than adults.

The application is supported by data from the phase 3 UNIFI Jr trial, which evaluated intravenous induction followed by randomized subcutaneous maintenance therapy through 52 weeks and demonstrated sustained clinical response consistent with adult UC findings. If approved, the indication would expand a therapy with an established safety and efficacy profile across multiple immune-mediated conditions and complement Johnson & Johnson’s parallel pediatric submission for Crohn’s disease, offering additional biologic options for young children with limited treatment choices.

2. FDA approves first treatment for thymidine kinase 2 deficiency

On November 3, 2025, the FDA approved Kygevvi (doxecitine and doxribtimine) powder for the treatment of thymidine kinase 2 deficiency (TK2d) in adults and in pediatric patients with symptom onset at or before 12 years of age, granting the therapy Breakthrough Therapy Designation. TK2d is an ultra-rare mitochondrial DNA maintenance disorder marked by progressive muscle weakness and respiratory failure, with only about 120 cases described in the literature.

The approval was supported by survival data from a phase 2 trial, retrospective chart reviews, and an expanded access program demonstrating markedly improved long-term outcomes—4% mortality in treated patients vs 36% in matched controls, with mean survival extended to 9.6 years compared with 5.7 years. Additional pharmacokinetic studies confirmed a significant food effect and supported a thrice-daily dosing regimen with meals. Kygevvi was generally well tolerated, offering the first FDA-approved treatment for a profoundly debilitating and historically underdiagnosed mitochondrial disorder.

3. FDA approves linaclotide (Linzess) for pediatric patients aged 7 years and older with IBS-C

On November 5, 2025, the FDA approved linaclotide (Linzess) capsules for children aged 7 years and older with irritable bowel syndrome with constipation (IBS-C), making it the first therapy authorized for this pediatric population. The approval was supported by a phase 3 randomized trial in 328 children that showed significant improvements in spontaneous bowel movement frequency, stool consistency, and early treatment response compared with placebo. Diarrhea was the most common adverse event, occurring in 4% of patients, with an overall safety profile consistent with adult data. The decision provides a long-needed, evidence-based option for managing chronic constipation and abdominal symptoms in children and adolescents, a population with limited approved treatments.

4. GeneDx receives FDA Breakthrough Device Designation for whole genome and exome tests

In late October, the FDA granted Breakthrough Device Designation to GeneDx’s whole genome (GenomeDx) and whole exome (ExomeDx) sequencing tests for diagnosing patients with life-threatening or genetically based diseases. The designation recognizes the potential of comprehensive genomic testing to accelerate diagnosis for individuals facing a years-long “diagnostic odyssey,” particularly those with rare or unexplained conditions.

Supported by GeneDx’s Infinity dataset—one of the largest rare-disease genomic resources, with nearly one million sequenced exomes/genomes and more than 7 million phenotypic datapoints—the tests are designed to detect both coding and noncoding variants that standard panels may miss. The Breakthrough designation provides an expedited FDA review pathway, underscoring the growing role of genomic analysis as a first-line diagnostic tool and advancing GeneDx’s goal of making precision medicine accessible across diverse patient populations.

5. FDA updates Elevidys labeling with boxed warning and revised indication following reports of fatal liver injury

On November 14, 2025, the FDA approved major labeling revisions for delandistrogene moxeparvovec-rokl (Elevidys), adding a Boxed Warning and narrowing its indication to ambulatory patients aged 4 years and older with a confirmed Duchenne muscular dystrophy (DMD) gene mutation. The update follows two fatal cases of acute liver failure and an additional severe liver injury in non-ambulatory children treated with the AAV-based gene therapy, prompting the removal of the non-ambulatory indication and the addition of new safety and monitoring requirements.

The revised label includes contraindications for specific exon deletions, cautions for patients with liver impairment or recent infection, and guidance for intensive post-infusion monitoring, including weekly liver function tests for at least three months. The FDA is also mandating a postmarketing observational study to further evaluate serious liver injury risk. These changes aim to improve clinical decision-making and enhance patient safety while maintaining Elevidys as a treatment option for a more narrowly defined DMD population.

6. FDA accepts roflumilast cream 0.3% sNDA to treat plaque psoriasis in children 2 to 5 years

On November 17, 2025, the FDA accepted the supplemental New Drug Application for roflumilast (Zoryve) cream 0.3%, advancing its potential to become the first topical PDE4 inhibitor approved for plaque psoriasis in children as young as 2 years, with a PDUFA action date of June 29, 2026. The filing is supported by a MUSE study in children aged 2–5 years and long-term open-label data demonstrating sustained efficacy, favorable tolerability, and minimal systemic exposure across pediatric age groups.

If approved, the once-daily, steroid-free cream—formulated without sensitizing excipients—would expand access for young children who often have disease in sensitive areas such as the face and intertriginous skin and currently have very limited FDA-approved treatment options. The potential age expansion represents a meaningful advance in pediatric dermatology, offering a gentler, effective alternative for a population with significant unmet therapeutic needs.

7. FDA restricts sale of unapproved ingestible fluoride drugs for children

On October 31, 2025, the FDA announced plans to restrict and remove unapproved ingestible fluoride prescription drug products intended for children, citing potential risks to the developing gut microbiome and associations with thyroid dysfunction. The action follows a complete safety review of pediatric fluoride exposure, during which the agency concluded that these products, never formally approved, pose unnecessary systemic risks. While topical fluoride remains supported, the FDA's move aligns with broader efforts to prioritize safe, evidence-based pediatric dental care and may significantly impact prescribing practices for low-risk children.

8. FDA expands antithrombin III (human) label to pediatric patients with rare blood-clotting disorder

On November 18, 2025, the FDA approved an expanded indication for antithrombin III (human) (THROMBATE III), authorizing its use in pediatric patients with hereditary antithrombin deficiency (hATd) and making it the first and only antithrombin concentrate approved for both adults and children with the disorder.

The decision was supported by extrapolated adult clinical trial data demonstrating that ATc can be safely and effectively used in pediatric patients, helping address a long-standing treatment gap for a population at high thrombotic risk—an estimated 85% of individuals with hATd experience at least one thrombotic event by age 50. The updated indication provides clinicians with a validated option for preventing and treating thromboembolic events surrounding surgery, childbirth, and other high-risk situations in children. Safety considerations include potential hypersensitivity reactions, infectious agent transmission due to plasma-derived manufacturing, and enhanced anticoagulant effects when used with heparin, with routine coagulation monitoring recommended.

9. FDA approves EYLEA HD injection 8 mg to treat macular edema following RVO and monthly dosing

On November 19, 2025, Regeneron Pharmaceuticals announced FDA approval of aflibercept 8 mg (Eylea HD) for treating macular edema following retinal vein occlusion (RVO) with dosing every 8 weeks after an initial monthly loading phase. The agency also approved a monthly dosing option for all other Eylea HD indications—wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy—for patients who may benefit from returning to more frequent dosing after extended intervals.

Designed to provide comparable efficacy to the original aflibercept with fewer injections, Eylea HD now offers physicians expanded flexibility with dosing intervals ranging from every 4 to 16 weeks, depending on indication and treatment response. Supported by more than a decade of aflibercept data and multiple pivotal trials, the updated approval enhances individualized care for patients with vision-threatening retinal disease.

10. FDA grants 510(k) clearance for Clarity algorithm to detect electrographic seizures in newborns

On November 24, 2025, CeriBell Inc. announced FDA 510(k) clearance of its Clarity algorithm, making the Ceribell System the first AI-powered point-of-care EEG technology cleared to detect electrographic seizures across all ages—from preterm newborns through adults. The decision was supported by EEG data from more than 700 patients, representing the largest validation dataset ever used for neonatal seizure detection.

Designed to address the critical gap in 24/7 EEG access in NICUs, Clarity provides rapid bedside neurodiagnostic assessment, helping identify seizures that otherwise go undetected—an issue affecting up to 90% of NICU patients without continuous monitoring. The clearance expands clinical access to real-time neurologic evaluation in the most vulnerable patients, aligning with growing recommendations for continuous EEG use in neonatal seizure management and supporting timely intervention to protect long-term neurodevelopmental outcomes.

11. FDA approves first gene replacement therapy (Itvisma) for patients 2 years and older with spinal muscular atrophy

On November 25, 2025, the FDA approved Itvisma (onasemnogene abeparvovec-brve) for children aged 2 years and older, adolescents, and adults with spinal muscular atrophy (SMA) caused by a confirmed SMN1 mutation, making it the first gene replacement therapy authorized for this broader SMA population. Administered as a single fixed intrathecal dose, Itvisma is designed to deliver a functional SMN1 gene and support sustained SMN protein expression, potentially reducing reliance on chronic therapies.

The approval was based on the phase 3 STEER trial, which showed significant and sustained motor-function improvement over 52 weeks, and the phase 3b STRENGTH study, which demonstrated motor-function stabilization in patients transitioning from nusinersen or risdiplam. With a safety profile consistent across studies and mostly mild respiratory and febrile adverse events, Itvisma offers a transformative treatment option for older patients with SMA—addressing a longstanding unmet need in a population with progressive disease and limited therapeutic alternatives.