Morgan Ebert, Executive Editor

A recap of the top clinical trial and pipeline data readouts affecting pediatric care during September 2026.

Upadacitinib showed continued facial and body repigmentation in NSV through week 76, with numerically higher responses when combined with NB-UVB.

A recap of the top FDA approvals/regulatory decisions/pipeline updates affecting pediatric care during September 2026.

Mavacamten becomes the first cardiac myosin inhibitor approved for adults and pediatric patients weighing 30 kg (66 lbs) or more with symptomatic oHCM.

In a 48-patient phase 1b trial, ecnoglutide was well tolerated in adolescents with obesity and reduced BMI up to 12.6% at 20 weeks.

DBV filed a BLA for the Viaskin peanut patch in peanut-allergic children aged 4-7, citing phase 3 VITESSE data, and requested priority review.

ScolimiR-3D, a 6-microRNA blood test to predict severe curve progression in AIS, received FDA Breakthrough Device designation.

Brian Vickery, MD, discusses a phase 1 trial evaluating the safety and tolerability of a microneedle stamp designed to deliver peanut protein through the skin.

The FDA approved tiratricol (Emcitate) for peripheral thyrotoxicosis in MCT8 deficiency, the first approved therapy for the rare disorder.

FDA accepts leniolisib sNDA with priority review for children with APDS aged 4+ weighing 13 to <27 kg; PDUFA date is January 30, 2027.

FDA approves oral ALK2 inhibitor zilurgisertib for FOP in patients 12+, based on phase 2 PROGRESS data on new heterotopic ossification volume.

FDA approved obinutuzumab for relapsing or steroid-dependent childhood-onset INS, based on INShore data showing 95.5% vs 73.2% sustained remission.

Glucommander Pediatrics received FDA Breakthrough Device Designation for IV insulin dosing in hospitalized children younger than 2 years, but remains uncleared.

In a Hirschsprung disease mouse model, diet shifted gut microbes and inflammation, with a 3-fold survival difference between standard diets.

A national claims analysis found 16.8% of pediatric GLP-1 users developed a nutritional deficiency within a year, most often vitamin D, while few received nutritional counseling.

Higher preconception maternal and paternal vitamin B12 was tied to fewer birth defects in a Shanghai cohort of 3032 couples.

Lavengratinib increased annualized height velocity in 7 children with achondroplasia, but open-label phase 2 findings remain preliminary.

The FDA granted satralizumab priority review for MOGAD after phase 3 findings showed a 68% reduction in relapse risk versus placebo.

Suicide-related pediatric ED visits rose 74% from 2016 to 2022; about half occurred at EDs with lower pediatric volume, a national analysis found.

The FDA accepted the dersimelagon NDA with priority review for EPP and XLP; a decision on the oral MC1R agonist is expected by February 2027.

FDA approved levacetylleucine (Aqneursa) for ataxia in ataxia-telangiectasia, the first therapy for the disease, in patients 15 kg or heavier.

The FDA granted priority review to efzimfotase alfa for HPP in patients aged 2 years and older; a decision is expected in the first half of 2027.

Adolescent VALIANT subgroup data show pegcetacoplan cut proteinuria 75% vs placebo, supporting its expanded pediatric kidney indication.

The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time AAV9 gene therapy, as the first treatment for pediatric MPS IIIA (Sanfilippo syndrome type A).

Aquestive resubmitted its NDA for dibutepinephrine sublingual film Anaphylm, citing new human factors and PK data addressing the FDA's CRL.

A Bifidobacterium-based synbiotic was well tolerated and increased colonization of administered strains in infants and toddlers.

Children with atopic dermatitis have higher ADHD risk, particularly with early, severe disease and sleep disturbance, a review found.

Baseline severity and clinical phenotype may help predict response to systemic therapy in children with alopecia areata.

Phase 3 CROSSING trial data show tezepelumab improved histologic remission and dysphagia versus placebo through week 52 in EoE.

FDA approved apitegromab-mstn (Isembyld) for SMA in patients 2 years and up, based on phase 3 data showing added motor gains with SMN2 therapy.