Drug Pipeline News

The approval marks the first treatment for this neurodegenerative with a low survival rate beyond 3 years.

The Phase 3 Beeline trial has begun dosing patients to evaluate radiprodil, a targeted NMDA receptor modulator designed to address the underlying biology of GRIN-related neurodevelopmental disorder.

Findings from a phase 2 trial showed that once-weekly TransCon CNP plus TransCon hGH produced durable growth and proportionality benefits in children with achondroplasia.

Results from a large randomized trial found that a midpregnancy blood test paired with targeted interventions improved neonatal outcomes in low-risk pregnancies.

FDA approved caplacizumab for pediatric patients ≥12 years with acquired TTP, supported by retrospective data showing 80% remission.

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

With the priority review, the Prescription Drug User Fee Act date is set for April 29, 2026, for potential approval in this younger indication.

The FDA approved narsoplimab-wuug as the first targeted treatment for transplant-associated thrombotic microangiopathy in adults and children.

Take a look back at the top 5 FDA approvals of 2025.

Approval of the expanded indication fullfilled all post-marketing requirements outlined in the original approval letter.

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.

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

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.

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.