Drug Pipeline News

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.

The investigational compound centanafadine to treat ADHD in children, adults, and adolescents, is a first-in-class NDSRI.

The clearnace was granted to the Ceribell System for electrographic seizure detection in newborns pre-term and older.

George Rodgers, III, MD, PhD, emphasized that antithrombin III’s expanded pediatric label provides "a sterile, plasma-derived product that will specifically correct the deficiency in a more safe fashion."

A phase 1b study of zelpultide alfa demonstrated a favorable safety profile and encouraging early reductions in BPD and ventilation duration, supporting advancement to a pivotal international Phase 2b/3 program.

The agency approved a monthly dosing option for select patients acorss all approved indications, including wAMD, DME, DR, and RVO.

“This label expansion helps close a long-standing gap in the treatment of pediatric patients with hereditary antithrombin deficiency,” said George M. Rodgers, III, MD, PhD.

The target action date for roflumilast cream 0.3% for plaque psoriasis in children 2 to 5 years is set for June 29, 2026.