Drug Pipeline News

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.

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.

Long-term data show sustained survival, immune recovery, and metabolic correction after lentiviral gene therapy for ADA-SCID, with no vector-related safety events.

FDA adds boxed warning to Elevidys and limits use to ambulatory DMD patients ≥4 years after reports of fatal acute liver failure in non-ambulatory patients.

Sub-analysis of ADORING trials shows VTAMA cream improved skin clearance, symptoms, and patient-reported outcomes in children aged 2-17.

Dupilumab maintained histologic and endoscopic improvements across all pediatric age groups with EoE, according to Joshua Wechsler, MD.

Thomas Wallach, MD, discusses phase 3 safety data of tenapanor in pediatric patients with IBS-C, presented at the 2025 NASPGHAN Annual Meeting.

Long-term analysis of the PEDFIC studies presented at NASPGHAN 2025 demonstrated sustained reductions in bile acids and pruritus with odevixibat in FIC1 deficiency.

At NASPGHAN 2025, investigators presented 52-week safety data from an ongoing phase 3 trial of linaclotide, the first approved treatment for patients aged 7 and older with IBS-C.

FDA approves first irritable bowel syndrome with constipation (IBS-C) treatment, linaclotide (Linzess), for children aged 7 years and older based on pediatric and adult trial data.

FDA approves Kygevvi, the first therapy for thymidine kinase 2 deficiency, a rare mitochondrial disease marked by muscle weakness and early death.