CRENESSITY (Crinecerfont)

July 1, 2026: Neurocrine Biosciences, Inc. (NASDAQ: NBIX) announced initiation of a Phase 2 open-label, single-arm clinical study of crinecerfont (CRENESSITY) in children aged 3 months to under 4 years with classic congenital adrenal hyperplasia (CAH). The 24-week trial will enroll 20 participants and has a primary objective of assessing safety and tolerability; secondary objectives include pharmacokinetics and pharmacodynamic effects on hormone biomarkers. The study is being conducted under an FDA Pediatric Written Request and is expected to support a planned supplemental New Drug Application (sNDA) to expand the U.S. label to patients under 4 years of age.

Separately, the company reported achieving target enrollment for a Phase 2 study in the European Union evaluating safety and tolerability of crinecerfont in children from birth to under 2 years with classic CAH.

The release notes crinecerfont was FDA-approved in 2024 as an adjunct to glucocorticoid replacement to control androgens in adult and pediatric patients aged 4 years and older with classic CAH.