In the explorer8 trial, concizumab-mtci significantly reduced annualized bleeding rates (ABR) by 86% in hemophilia A and 79% in hemophilia B patients compared with no prophylaxis. The therapy is administered as a daily subcutaneous injection and was generally well tolerated, with injection site reactions and headache being the most common adverse events.
Click here for the full approval details.
FDA news in pediatrics: July 2025
Another month has come and gone, and with it, several FDA approvals and regulatory updates related to the ever-evolving landscape of pediatric health care.
In our July monthly recap, we list our top FDA-related news items in a quick, easy-to-read format so you can stay informed.
Take a look at our detailed coverage of FDA-related news from July, and stay in touch with our digital newsletters, which provide practical information for today's pediatrician.
Click here to view our complete July FDA recap and see the top headlines from the month.
CDC: Kindergarten vaccination decreases, exemptions increase
Vaccination coverage among kindergartners in the United States declined during the 2024–2025 school year while exemption rates rose, according to new findings from the CDC. Data from states and the District of Columbia showed decreases in coverage for all routine childhood vaccines compared to the previous year. Most states also reported year-over-year declines in protection against illnesses such as measles, polio, and varicella.
Click here for the CDC's latest vaccination data.
Richard Auchus, MD: Crinecerfont's positive weight-related outcomes in children with CAH
Twelve-month data from the phase 3 CAHtalyst pediatric trial, presented at ENDO 2025, showed that crinecerfont (Crenessity; Neurocrine Biosciences), when added to glucocorticoid therapy, improved weight-related outcomes in children with classic congenital adrenal hyperplasia (CAH). The trial demonstrated a reduction in adrenal androgens, enabling lower glucocorticoid doses and a decrease in body mass index (BMI) among participants receiving crinecerfont.
Richard Auchus, MD, PhD, professor of pharmacology and internal medicine at the University of Michigan, highlighted the metabolic risks these patients face. “Children with CAH have a high prevalence of obesity, as do adults with CAH, which places them at increased risk for cardiovascular disease,” he explained. "In adults, secondary to adrenal crisis, the second most common cause of death in CAH is cardiovascular disease. They have a significant metabolic risk, which begins developing in childhood due to prolonged exposure to the high doses of glucocorticoids traditionally necessary to manage the condition adequately."
Click here to watch the full interview with Auchus.