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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, May 5, to Friday, May 9, 2025), and click on each link to read and watch anything you may have missed.
On May 7, 2025, ARS Pharmaceuticals announced that neffy, the epinephrine nasal spray, is now available by prescription in the US for children aged ≥4 years who weigh between 33 and <66 lbs to treat Type I allergic reactions, including anaphylaxis. The announcement follows the March 6, 2025 FDA approval expanding neffy’s indication to this younger weight group, making it the first and only needle-free epinephrine treatment for children under 66 lbs. Originally approved on August 9, 2024, for children weighing ≥66 lbs, neffy offers a portable, easy-to-use alternative to epinephrine auto-injectors, with no needle and demonstrated ease-of-use in human factor studies. It also has a 24-month shelf life and remains stable for 3 months at temperatures up to 122°F. Approval was supported by pharmacokinetic and pharmacodynamic data showing bioequivalence to injectable epinephrine in adults and children.
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The case involves an 18-day-old female presenting to the emergency department with intermittent cyanosis, suspected apnea, decreased feeding, and a history of retractions, nasal flaring, and grunting. Vital signs revealed hypoxemia and tachypnea, with oxygen saturation levels fluctuating between 65% and 100% on room air. A chest X-ray image was provided for diagnostic evaluation. Readers are encouraged to vote on the most likely diagnosis and return to the site for the full case details, differential diagnoses, and final diagnosis.
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In this interview with Suzanne Hollander, MS, RD, LDN, of Boston Children’s Hospital, a discussion of treatment advances in phenylketonuria (PKU), a rare metabolic disorder. Hollander explained that untreated PKU leads to neurocognitive and mood complications due to toxic levels of phenylalanine (Phe). The current standard of care includes a strict Phe-restricted diet paired with medical formula, posing significant challenges. Hollander highlighted sepiapterin, an investigational therapy shown in the Phase 3 APHENITY trial to lower blood Phe levels and enable dietary liberalization. In the trial, 84% of participants achieved target Phe levels, with many doubling or tripling their natural protein intake and reducing reliance on formula. The FDA is expected to review sepiapterin by July 29, 2025.
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On May 7, 2025, Infinant Health announced that the FDA granted orphan drug and rare pediatric disease designations to INF108, an investigational therapy for the prevention of necrotizing enterocolitis (NEC) in preterm infants. NEC is a serious gastrointestinal condition and a leading cause of mortality in neonatal intensive care units, with an estimated incidence of 7% among very low birth weight infants, according to a 2020 meta-analysis. The orphan designation supports development of therapies for rare diseases, while the rare pediatric disease designation makes INF108 eligible for a priority review voucher upon potential FDA approval. Infinant Health plans to begin clinical trials of INF108 in preterm infants.
In a video interview with Contemporary Pediatrics, Peter S. Jensen, MD, discusses why unmonitored social media use among youth is a growing mental health concern—and what pediatricians, parents, and policymakers must do to address it. “We need a learner’s permit model,” says Jensen.
