Drug Pipeline News

If approved, the expanded indication would make roflumilast cream 0.05% the first once-daily, steroid-free topical PDE4 inhibitor approved specifically for this age group.

FDA cleared an IND for a NCATS-sponsored AAV9/SUMF1 gene therapy in multiple sulfatase deficiency, moving the ultra-rare disease toward its first-in-human trial.

FDA accepted Pharvaris's NDA for deucrictibant IR, an oral B2 antagonist for HAE attacks, based on phase 3 RAPIDe-3 data.

A look back at the FDA approvals, regulatory decisions, and pipeline updates in the pediatric health care space from June 2026.

A look back at the latest clinical trial updates for the pediatric population during June 2026.

FDA expands approval of roflumilast cream 0.3% for plaque psoriasis to children aged 2 years and older, offering the first topical PDE4 inhibitor.

A look back at the latest clinical trial updates for the pediatric population during the second quarter (April-June) of 2026.

FDA approved risankizumab for children 6 and older with plaque psoriasis or psoriatic arthritis, adding a weight-based 55-mg syringe for those under 40 kg.

A look back at the FDA approvals, label expansions, and regulatory decisions in the pediatric health care space from April through June 2026.

A preview of 5 pending FDA decisions for pediatric patients expected in the second half of 2026, spanning ADHD, psoriasis, a metabolic gene therapy, cardiomyopathy, and muscular dystrophy.

BM-3103 received FDA Fast Track designation for epidermolysis bullosa simplex as a phase 2 trial evaluates safety and efficacy.

Teva's NDA for ecopipam rests on phase 3 data showing a 50% relapse-risk cut, potentially the first new pediatric Tourette mechanism in a decade.

FDA approved the first generic rifapentine for active and latent tuberculosis, expanding treatment options for eligible pediatric patients.

In a parallel regulatory action, the European Medicines Agency has also validated a marketing application for Tivicay covering the neonatal indication.

New FDA-approved options for molluscum contagiosum—in-office cantharidin and at-home berdazimer gel—reduce stigma and avoid painful removal.

Cadrenal has announced that it plans to seek FDA Rare Pediatric Disease Designation for tecarfarin in children with Kawasaki disease and coronary aneurysms.

Baloxavir marboxil is a first-in-class cap-dependent endonuclease inhibitor that functions as a prodrug.

The approval is based on data from the phase 3 STRIDE-13 trial.

BioMarin reports 3-year vosoritide growth data in hypochondroplasia and early BMN 333 findings in achondroplasia.

pCPA gains FDA Orphan Drug Designation for monoamine oxidase deficiency, signaling early development for an ultrarare neurodevelopmental disorder.

Diazoxide choline showed sustained hyperphagia improvements in Prader-Willi syndrome after randomized withdrawal and retreatment.

Setmelanotide reduced BMI measures, fat mass, and hyperphagia scores in interim phase 2 Prader-Willi syndrome data.

The FDA has approved gadoquatrane (Ambelvist), a next-generation macrocyclic GBCA that delivers effective CNS and whole-body MRI contrast at 60% less gadolinium than standard macrocyclic agents.

The FDA granted accelerated approval to teplizumab (Tzield) for children aged 8–17 with recently diagnosed stage 3 T1D, based on PROTECT phase 3 data.

The integrated CGM, first cleared over-the-counter for adults in 2024, is now available without a prescription for people 2 years of age and older who do not use insulin.