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Thank you for visiting the Contemporary Pediatrics® website. Take a look at some of our top stories from the week (Monday, March 24, to Friday, March 28, 2025), and click on each link to read and watch anything you may have missed.
On February 18, 2025, Solid Biosciences announced positive interim data for SGT-003, a gene therapy candidate for Duchenne muscular dystrophy (DMD). In the phase 1/2 INSPIRE DUCHENNE trial, the first three patients showed an average microdystrophin expression of 110% and improvements in multiple muscle health biomarkers. Craig McDonald, MD, an investigator in the trial, emphasized the potential of SGT-003 to enhance muscle integrity and cardiac function, with no serious adverse events reported so far.
On March 26, 2025, the FDA approved VYKAT XR (diazoxide choline extended-release tablets) for hyperphagia in Prader-Willi syndrome (PWS) patients aged 4 years and older. This marks the first approved treatment targeting the hallmark symptom of PWS—persistent, uncontrollable hunger. In phase 3 trials, VYKAT XR demonstrated significant improvements in hyperphagia, aggressive behavior, and metabolic parameters. Advocates say the approval represents a major step forward for PWS treatment.
The FDA has approved fitusiran (Qfitlia; Sanofi) as a routine prophylaxis for hemophilia A and B patients aged 12 years and older. The treatment, administered subcutaneously once every two months, works by reducing antithrombin levels to increase clotting ability. Phase 3 trials showed a 71%–73% reduction in annualized bleeding rates. The approval includes a boxed warning for thrombotic events, gallbladder disease, and liver toxicity.
The FDA has approved gepotidacin (Blujepa; GSK), for use in treating uncomplicated urinary tract infections (uUTIs) in female patients aged at least 12 years and weighing at least 40 kg. Throughout their lifetime, over 50% of women experience uUTIs, with recurrent infection reported in approximately 30%.1 This can lead to discomfort and restriction of daily activities, posing a significant patient burden.
Several key pediatric FDA decisions are expected in the second quarter of 2025, potentially reshaping treatment options for children with a range of conditions. From dermatologic diseases to infectious diseases, upcoming regulatory actions could bring significant advancements in pediatric care. This article highlights five anticipated FDA decisions based on PDUFA dates and ongoing clinical developments.
Fill out some basic information at the link below, and view our list of key potential FDA approvals on deck for the second quarter of 2025.
A 13-year-old female with a history of iron deficiency anemia presented with altered mental status preceded by 5 days of headache, vomiting, nausea, and worsening somnolence that was not responsive to flumazenil, naloxone, or ammonia salts. She had a Glasgow coma scale of 12 and was drowsy with no cranial nerve deficits, or other abnormal physical examination findings. Laboratory evaluation showed microcytic anemia (hemoglobin 7.7 g/dL and MCV (mean corpuscular volume):64.7, lymphocytosis, and mildly elevated liver enzymes [Aspartate Aminotransferase (AST) 52, Alanine Transaminase (ALT) 110] otherwise her electrolytes and kidney function were normal.
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