Drug Pipeline News

Observations were not specific to apitegromab, and the CRL did not cite any other approvability concerns related to efficacy and safety data.

The FDA announces significant updates on acetaminophen risks in pregnancy and approves leucovorin calcium for treating CFD in children.

Zilganersen met the primary endpoint of gait stabilization, showing statistically significant benefit on gait speed and favorable safety.

The planned phase 2 study of SAT-3247 will evaluate safety, tolerability, and functional outcomes in children with Duchenne muscular dystrophy.

The federal agency granted accelerated approval to elamipretide HCl to treat patients with Barth syndrome.

Rezpegaldesleukin improved disease severity and patient-reported outcomes in a phase 2b atopic dermatitis trial with sustained benefit.

The Phase 3 SURPASS-PEDS trial found tirzepatide improved glycemic control and weight outcomes in youth-onset type 2 diabetes, with benefits lasting one year.

In this video interview, John Browning, MD, comments on the recent pediatric indication approved for ruxolitinib to treat atopic dermatitis.

New phase 3 data show icotrokinra maintained high PASI90 responses in pediatric and adult patients at 1 year.

The updated label is based on real-world data collected since B-VEC was launched in the United States, following its approval in May 2023.

According to new, real-world data for trofinetide, caregivers reported quality-of-life, alertness, and interaction improvements at 12 months of treatment.

Get caught up with Contemporary Pediatrics! This list helps you navigate our top stories from the week, all in one place.

The FDA approves recombinant VWF for pediatric von Willebrand disease, enhancing treatment options for children during bleeding episodes and surgeries.

Watch our FDA pipeline news recap for the month for August 2025, as well as a preview of PDUFA dates listed for September.

Arcutis has submitted an sNDA to the FDA for roflumilast cream 0.3% for plaque psoriasis in children aged 2 to 5 years.

A look back at the FDA submissions and regulatory decisions in the pediatric health care space from August 2025.

In recent studies, donidalorsen (Dawnzera) has demonstrated durable efficacy and long-term disease control.

High-dose centanafadine significantly improved ADHD symptoms in children aged 6 to 12 years and was well tolerated, according to findings from a phase 3 clinical trial.

If approved, vatiquinone would have been the first therapy approved for pediatric patients with FA.

Richard Auchus, MD, PhD, discusses how crinecerfont is improving CAH care and easing the transition from pediatric to adult management.

FDA approves first generic iron sucrose injection for treating iron deficiency anemia in CKD patients 2 years and older.

Brensocatib was granted FDA priority review in February 2025 for the small molecule, oral, reversible inhibitor.

FDA accepts priority review of gepotidacin, a first-in-class oral antibiotic, for treating uncomplicated urogenital gonorrhea in patients ≥12 years.

Manufacturer MannKind has submitted a supplemental Biologics Application to the FDA for its inhaled insulin Afrezza.

The expanded label of tocilizumab-anoh now includes treatment for patients aged 2 years and older with cytokine release syndrome (CRS).