Get caught up with Contemporary Pediatrics! This list helps you navigate our top stories from the week, all in one place.
Thank you for visiting the Contemporary Pediatrics® website. Take a look at some of our top stories from last week (Monday, December 8, to Friday, December 12, 2025), and click on each link to read and watch anything you may have missed.
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On December 8, 2025, the FDA approved omidubicel-onlv (Omisirge) as the first hematopoietic stem cell transplant therapy authorized to treat patients with severe aplastic anemia, expanding options for adults and children aged 6 years and older who lack a compatible donor. The cord blood–derived therapy is designed to accelerate blood and immune recovery following transplant and represents a new graft option for a rare, life-threatening condition with limited treatment alternatives.
On December 9, 2025, the FDA approved Waskyra (etuvetidigene autotemcel) as the first cell-based gene therapy in the United States for Wiskott-Aldrich syndrome, a rare and life-threatening immunodeficiency. The one-time therapy uses a patient’s own genetically modified hematopoietic stem cells and is indicated for pediatric patients aged 6 months and older and adults who lack a suitable matched donor, marking a major advance for a population with historically limited treatment options.
On December 11, 2025, the FDA approved a supplemental new drug application for gepotidacin (Blujepa; GSK) as an oral treatment for uncomplicated urogenital gonorrhea in patients aged 12 years and older weighing at least 45 kg who have limited or no alternative treatment options. The decision introduces the first noninjectable therapy for gonorrhea in the United States in more than three decades, offering a new oral option amid rising case counts and growing concerns about antimicrobial resistance.
On December 12, 2025, the FDA approved BioCryst’s berotralstat (Orladeyo) oral pellets for prophylaxis in pediatric patients aged 2 to 11 years with hereditary angioedema (HAE), making it the first targeted oral preventive therapy available for children younger than 12 years with the condition. The approval expands treatment options for a rare, potentially life-threatening disease that often begins in early childhood and can significantly affect quality of life for patients and families.
On December 12, 2025, the FDA approved trofinetide (Daybue Stix; Acadia Pharmaceuticals), a powder formulation for oral solution, for the treatment of Rett syndrome in adult and pediatric patients aged 2 years and older. The dye- and preservative-free formulation provides greater flexibility in dosing volume and taste while maintaining the same efficacy and safety profile as the original Daybue oral solution, which remains available as the first and only FDA-approved treatment for Rett syndrome.
On December 12, 2025, the FDA approved Nuzolvence (zoliflodacin; Innoviva Specialty Therapeutics), a single-dose oral antibiotic for the treatment of uncomplicated urogenital gonorrhea in adults and pediatric patients aged 12 years and older who weigh at least 77 pounds. The approval marks one of the first new treatment options for gonorrhea in nearly two decades and provides a noninjectable alternative amid rising infection rates and growing antimicrobial resistance.
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