Drug Pipeline News

Ferric maltol is now an FDA-approved oral iron therapy for both adults and adolescents with iron deficiency, aged 10 years and older.

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

Once-weekly navepegritide improved growth velocity, skeletal alignment, and functional outcomes in children with achondroplasia, with a favorable safety profile and potential implications for long-term care.

The FDA approved fibrinogen, human-chmt to treat acute bleeding episodes in adult and pediatric patients with congenital fibrinogen deficiency.

Abbott received FDA clearance for a new delivery system designed to simplify PDA closure in premature infants weighing as little as 2 lb.

Phase 3 data show the Viaskin peanut patch improved desensitization vs placebo in children aged 4 to 7 years, supporting a planned FDA BLA in 2026.

The SWIFT-1 and -2 phase 3 trials demonstrated "significantly" lower rate of annualized asthma exacerbations in patients receiving twice-yearly depemokimab vs placebo.

GeneDx’s ExomeDx and GenomeDx tests, now FDA Breakthrough Designated, offer comprehensive genomic analysis to improve diagnosis of rare and serious diseases.

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

“Having a drug that [patients] can be confident in that will be highly effective as well as well tolerated is a great advantage," said Edward Hook, MD.

The FDA has approved Nuzolvence as a new single-dose oral option for uncomplicated urogenital gonorrhea in adults and children 12 years and older who weigh at least 77 pounds.

Clinicians now have the first oral alternative to injectable therapy for gonorrhea, a shift Angela Hasler, APRN, CPNP-PC says could ease treatment barriers and improve public health outcomes.

The FDA approved Daybue Stix, a powder formulation of trofinetide, for Rett syndrome in patients 2 years or older.

Berotralstat becomes the first targeted oral prophylactic therapy for patients with HAE 2 years or older.

Phase 3 results show oral zoliflodacin was noninferior to ceftriaxone and azithromycin for uncomplicated urogenital gonorrhea.

This approval of gepotidacin is the first in a new antibiotic class for the treatment of gonorrhoea approved in over 3 decades.

Waskyra becomes the first FDA-approved gene therapy for Wiskott-Aldrich syndrome, offering hope to patients with this rare immunodeficiency.

The FDA approved omidubicel on December 8, 2025, the same day parent company Ayrmid announced positive results at the 2025 American Society of Hematology Annual Meeting and Exposition.

Marstacimab’s once-weekly subcutaneous administration demonstrated clinically meaningful superiority in key bleeding outcomes for adolescents and adults with hemophilia A or B.

FDA approves expanded indication for MED-EL cochlear implants, allowing use in infants 7 months or older with bilateral SNHL and broadening pediatric eligibility.

New US modeling study results find nirsevimab would avert the most infant RSV-LRTD events and hospitalizations compared with clesrovimab or RSVpreF maternal vaccination.

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

“It represents the first time an in vivo HSC-directed gene insertion therapy has been evaluated in a patient," said CEO Jim Burns in a statement.

A phase 2b randomized trial found once-weekly navepegritide improved growth velocity, skeletal alignment, and physical functioning in children with achondroplasia.