Drug Pipeline News

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).

The indication approved is for children aged 6 years and older who weigh at least 45 kg (99 lbs) for the preventive treatment of episodic migraine.

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

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

The FDA approved concizumab-mtci (Alhemo; Novo Nordisk) for hemophilia A/B without inhibitors, offering once-daily subcutaneous prophylaxis that significantly reduces bleeding rates.

Nektar Therapeutics remains on track to announce top-line phase 2 data in December.

Pegcetacoplan (Empaveli; Apellis Pharmaceuticals) is indicated for patients 12 years or older to reduce proteinuria.

Suzanne Hollander, MS, RD, LDN; and Timothy Tramontana, MD, offer thoughts on newly FDA-approved sepiapterin for PKU.

“Without treatment, individuals with PKU would have very high blood Phe levels...so that results in neurocognitive deficits and mood disorders," Suzanne Hollander, MS, RD, LDN, said.

The FDA has approved avatrombopag and new sprinkle formulation for children ≥1 year with chronic ITP, offering an oral, food-flexible treatment option.