5 FDA decisions to watch for during 2H 2026

After a first half of 2026 that brought historic firsts for Menkes disease, arginase 1 deficiency, achondroplasia, plaque psoriasis, and the neurologic complications of Hunter syndrome, the FDA's pediatric pipeline shows no signs of slowing. The back half of the year carries a slate of priority review decisions spanning neurodevelopmental, cardiac, metabolic, and neuromuscular disease—several of which could establish the first pharmacologic options ever available to the children and adolescents they're intended to treat.

The following is a preview of 5 notable FDA decisions on the horizon for pediatric patients in the second half of 2026, based on currently disclosed Prescription Drug User Fee Act (PDUFA) target action dates. As with any pending application, these dates are subject to change, and the FDA may approve, request more information on, or decline any of these therapies.

1. Centanafadine (Otsuka Pharmaceutical)

PDUFA date: July 24, 2026

Indication: Attention-deficit/hyperactivity disorder (ADHD) in children, adolescents, and adults

Background: Centanafadine is a first-in-class norepinephrine, dopamine, and serotonin reuptake inhibitor submitted for priority review in November 2025. The new drug application is supported by 4 pivotal phase 3 trials, including a dedicated pediatric study (NCT05428033) in children aged 6 to 12 years and an adolescent study (NCT05257265) in patients aged 13 to 17 years, both of which demonstrated statistically significant improvements on the ADHD Rating Scale-5 compared with placebo.¹ If approved, the once-daily capsule would introduce a novel nonstimulant mechanism for a condition affecting an estimated 6 million children and adolescents in the US, with a reported low potential for abuse or dependence.

2. Roflumilast cream 0.3% (Zoryve; Arcutis Biotherapeutics)

PDUFA date: June 29, 2026

Indication: Plaque psoriasis in children 2 to 5 years old

Background: This supplemental application seeks to extend the currently approved indication for roflumilast cream—available since 2022 for patients 6 years and older—down to children as young as 2. The filing is supported by a 4-week maximal usage systemic exposure study showing minimal-to-no systemic absorption, along with long-term open-label data demonstrating sustained efficacy and tolerability.² If cleared, it would become the first topical phosphodiesterase-4 inhibitor indicated for plaque psoriasis in this very young age group, offering a steroid-free option for disease that often involves sensitive facial and intertriginous skin.

3. DTX401/pariglasgene brecaparvovec (Ultragenyx Pharmaceutical)

PDUFA date: August 23, 2026

Indication: Glycogen storage disease type Ia (GSDIa)

Background: DTX401 is an investigational AAV8 vector gene therapy designed to deliver a functional copy of the G6PC gene, which is deficient in GSDIa, a metabolic disorder typically diagnosed in infancy that forces patients into a lifelong regimen of frequent cornstarch dosing to avoid severe hypoglycemia. The biologics license application, granted priority review, draws on data from 52 treated patients with up to 6 years of follow-up, including the randomized, placebo-controlled phase 3 GlucoGene trial, and reported reduced cornstarch requirements alongside maintained glucose control.³ If approved, it would be the first therapy to address the underlying genetic cause of GSDIa rather than its downstream metabolic consequences.

4. Mavacamten (Camzyos; Bristol Myers Squibb)

PDUFA date: September 30, 2026

Indication: Symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adolescents 12 to younger than 18 years

Background: Mavacamten, a cardiac myosin inhibitor approved for adults with oHCM since 2022, is under priority review for an adolescent indication based on the phase 3 SCOUT-HCM trial, which the company reports met its primary endpoint with an adolescent safety profile similar to that observed in adults.⁴ No pharmacologic therapy is currently approved for oHCM in this age group, where management has relied on beta-blockers and invasive septal reduction procedures that carry their own risks in younger patients. If cleared, mavacamten would become the first cardiac myosin inhibitor available to adolescents with this disease.

5. BBP-418 (BridgeBio Pharma)

PDUFA date: Anticipated late 2026 (NDA submission planned for 2026)

Indication: Limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) in patients 12 years and older

Background: LGMD2I/R9 is a progressive, autosomal recessive muscular dystrophy that often manifests in late childhood and has no FDA-approved treatment. BBP-418, an oral substrate supplementation therapy, is being evaluated in the phase 3 FORTIFY trial, whose interim analysis showed a statistically significant 1.8-fold increase in glycosylated alpha-dystroglycan and an 82% reduction in serum creatine kinase relative to placebo at 12 months, alongside early separation from placebo on a 100-meter timed ambulation test.⁵ BridgeBio has said it intends to submit a new drug application in 2026 and is targeting a launch in late 2026 or early 2027. If approved, it would be the first treatment for any form of limb-girdle muscular dystrophy.

References

1. Otsuka Pharmaceutical. Otsuka Pharmaceutical submits New Drug Application to U.S. FDA for centanafadine for the treatment of ADHD in children, adolescents, and adults. News release. November 25, 2025. Accessed June 26, 2026. https://www.otsuka-us.com/news/otsuka-pharmaceutical-announces-positive-topline-results-two-pivotal-phase-3-trials

2. Arcutis Biotherapeutics. FDA accepts supplemental New Drug Application for Arcutis' Zoryve (roflumilast) cream 0.3% for the treatment of plaque psoriasis in children ages 2 to 5. News release. November 17, 2025. Accessed June 26, 2026. https://www.arcutis.com/fda-accepts-supplemental-new-drug-application-for-arcutis-zoryve-roflumilast-cream-0-3-for-the-treatment-of-plaque-psoriasis-in-children-ages-2-to-5/

3. Ultragenyx Pharmaceutical Inc. Ultragenyx announces U.S. FDA acceptance and priority review of the Biologics License Application (BLA) for DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa). News release. February 23, 2026. Accessed June 26, 2026. https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-us-fda-acceptance-and-priority-review

4. Bristol Myers Squibb. U.S. Food and Drug Administration accepts for priority review Bristol Myers Squibb's supplemental New Drug Application for Camzyos (mavacamten) to treat adolescents with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). News release. 2026. Accessed June 26, 2026. https://news.bms.com/news/corporate-financial/2026/U-S--Food-and-Drug-Administration-Accepts-for-Priority-Review-Bristol-Myers-Squibbs-Supplemental-New-Drug-Application-for-Camzyos-mavacamten-to-Treat-Adolescents-with-Symptomatic-Obstructive-Hypertrophic-Cardiomyopathy-oHCM/default.aspx

5. BridgeBio Pharma, Inc. BridgeBio reports positive phase 3 results for small molecule BBP-418 in LGMD2I/R9 FORTIFY study. News release. October 27, 2025. Accessed June 26, 2026. https://investor.bridgebio.com/news/news-details/2025/BridgeBio-Reports-Positive-Phase-3-Results-for-Small-Molecule-BBP-418-in-LGMD2IR9-FORTIFY-Study/default.aspx