FDA news in pediatrics: April 2025
A look back at the FDA submissions and regulatory decisions in the pediatric health care space from April 2025.
Full recap from the 2025 Pediatric Academic Societies meeting
In this article, we recap our top stories, expert interviews, and Q+A discussions from the 2025 Pediatric Academic Societies meeting.
Uncovering the pediatrician's role in OTC STI testing, with Lea Widdice, MD
Lea Widdice, MD, details her recent presentation at PAS 2025 on OTC STI testing within pediatrics.
Elamipretide to treat Barth syndrome sees FDA target action delay
Stealth BioTherapeutics' CEO stated, "We hope to gain more information on the revised action date in the coming days."
FDA approves nipocalimab-aahu to treat generalized myasthenia gravis
The human FcRn-blocking monoclonal antibody is indicated for gMG patients aged 12 years and older.
Zika-based therapy for metastatic neuroblastoma with Tamarah Westmoreland, MD and Joseph Mazar, PhD
A PAS 2025 study by Mazar and Westmoreland shows Zika virus can eliminate both local and distant tumors in neuroblastoma mouse models.
Maternal anemia in early pregnancy linked to higher risk of congenital heart disease in offspring
A UK study links maternal anemia in early pregnancy with increased congenital heart disease risk in offspring.
FDA approves pz-cel for recessive dystrophic epidermolysis bullosa
The decision makes pz-cel the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with RDEB.
Necessity for iron deficiency screenings at birth with Geetika Kennady, MD, FAAP
New research led by Geetika Kennady, MD, FAAP, showed that 10.6% of newborns have iron deficiency, supporting a push for earlier screening.
Jillian Cotter, MD, MSCS, on recent advances in pediatric pneumonia management
"We've updated our internal clinical pathways to recommend 5 days for kids hospitalized with uncomplicated pneumonia," Cotter noted.
Youth cannabis use and depression, suicidal behavior, with Jesse Hinckley, MD, PhD
At PAS 2025, Jesse Hinckley, MD, PhD, presented data showing cannabis use sharply increases odds of depression and suicidal behavior in youth.
Artificial intelligence improves accuracy in estimating child abuse
New model refines prevalence estimates, reduces misdiagnoses compared to the current coding system.
Buprenorphine linked to improved outcomes for pregnant women with OUD and their infants
A recent study found reduced preterm birth, complications, and NICU use in large Tennessee Medicaid cohort.
Validating a digital stethoscope for pediatric breath sounds, with Jennifer Nestor, MD
Jennifer Nestor, MD, evaluated a digital stethoscope to validate pediatric breath sound recordings for potential machine learning algorithm development.
Parents more likely to ask about guns in homes when they receive firearm safety information
Study finds education from multiple sources increases parent inquiry about firearms.
Youth with neurodevelopmental disabilities face higher risk of poor cardiovascular health
New data from national survey highlights disparities and need for focused prevention strategies.
Pegtarazimod data confirms inflammation biomarkers in hypoxic ischemic encephalopathy infants
In addition, data presented at PAS revealed a predictable pharmacokinetic profile in the newborn population, consistent with adult humans.
Decreasing antibiotics for acute otitis media and community acquired pneumonia, with Elysha Pifko, MD
Shorter antibiotic courses for AOM and CAP in children proved effective across a pediatric health system, according to data presented by Elysha Pifko, MD.
Antibiotic use before age 2 linked to higher childhood BMI
Early antibiotic use before age 2 is linked to higher childhood obesity risk, with timing playing a key role in long-term weight outcomes.
New protocol accurately diagnoses pediatric anesthetic allergy
The study involved 88 pediatric patients, with 12.5% showing positive intradermal test results and 1.1% positive subcutaneous provocation.
FDA announces measures to phase out petroleum-based food dyes
One step will be a partnership with the National Institutes of Health to research how food additives impact children's health.
PNP perspective: Nasal epinephrine spray approved for children as young as 4 years
Donna Hallas, PhD, PPCNP-BC, CPNP, PMHS, FAANP, FAAN, shares her thoughts on the latest print issue of Contemporary Pediatrics
Confirming celiac disease is getting easier
"We are getting away from biopsies to diagnose CD, which makes diagnosis even easier, said Jon Matthew Farber, MD.
Poll shows many parents unsure when to talk to kids about puberty
Most want to start the conversation, but confidence and timing vary.
Sensory play for children with autism, with Joshua Feder, MD
Joshua Feder, MD, emphasizes that sensory play is most effective when rooted in relational strategies that support autistic children in daily environments.
New blood test predicts preterm birth risk early in pregnancy
A new study found that cell-free DNA from routine prenatal testing can accurately identify preterm birth risk without added cost or procedures.
Do probiotics reduce allergy symptoms? It’s complicated!
In 3 studies evaluating the effects of probiotics on eczema, probiotics groups reported a greater reduction in symptoms than placebo groups, but only when the allergy was IgE mediated.
Weekly review: FDA approves dupilumab, ACIP meets, and more
From the FDA approvals of dupilumab and maralixibat tablets to a new puzzler case study, get caught up with our weekly review article.
FDA issues CRL for aflibercept 8 mg extended dosing for all indications
The CRL issued by the FDA to Regeneron for extended EYLEA HD dosing to 24 weeks cited no safety or efficacy concerns.
The crucial role pediatricians play in early autism diagnosis and why it matters
Find out how pediatricians play a crucial role in early autism diagnosis and how to advise families post-diagnosis.
