Neurocrine Biosciences expands crinecerfont trials to infants

The study, conducted under an FDA Pediatric Written Request, will track 20 participants over a 24-week period. Researchers are focused on assessing how the drug performs in this vulnerable demographic, specifically monitoring its impact on hormone biomarkers and pharmacokinetics. Currently, there are no approved therapies for children under 4 years old, leaving many infants reliant on supraphysiologic doses of glucocorticoids—a treatment path often linked to metabolic issues, stunted growth, and cardiovascular risks.

Sanjay Keswani, Chief Medical Officer at Neurocrine Biosciences, noted that the trial is driven by the urgent need to reduce long-term dependence on high-dose steroids. By targeting the source of excess androgens through a non-glucocorticoid mechanism, the company hopes to provide a safer alternative for patients during their most sensitive developmental years. The data gathered from this trial will support a supplemental New Drug Application intended to broaden the U.S. indication for the drug, currently marketed as CRENESSITY. Parallel efforts are already underway in the European Union, where the company has reached target enrollment for a similar study involving children from birth to age 2.