Richard Auchus, MD, on how crinecerfont is changing CAH treatment

Since its FDA approval on December 13, 2024, crinecerfont (Crenessity; Neurocrine Biosciences) has brought new momentum to the management of classic congenital adrenal hyperplasia (CAH) in children and adolescents. The drug is approved for use alongside glucocorticoids in patients aged 4 years and older to reduce excessive adrenal androgen production, enabling lower steroid doses while maintaining hormonal control.¹

Richard Auchus, MD, PhD, professor of pharmacology and internal medicine at the University of Michigan, said the treatment offers a much-needed option for pediatric endocrinologists who struggle to control the condition without risking long-term harm from high-dose steroids.

“If they use a higher dose of glucocorticoid, not only will the patient get obese, but they’re going to suppress their growth, they’re going to interfere with their sleep and their pubertal development. They have another option now that is not going to put them at risk for these things,” he explained.

Auchus noted the approval has sparked “a lot of excitement in the adult community” as well. By enabling a “block-and-replace” approach—suppressing androgens with crinecerfont and replacing cortisol separately—the therapy could encourage more adult endocrinologists to take on CAH care and improve transitions from pediatric to adult management. Audience polling at a recent Endocrine Society meeting revealed that at least 50% of children with CAH do not transition smoothly to adult care, a gap Auchus called “very unacceptable.”

WATCH MORE: Richard Auchus, MD: Crinecerfont's positive weight-related outcomes in children with CAH²

While not every child will require crinecerfont, Auchus stressed its potential for adolescents with difficult-to-control disease. For middle school and high school students, multiple daily hydrocortisone doses can be burdensome, but the new therapy may reduce the need for late-day or bedtime doses. Pediatricians also play a key role as part of the care team.

“You’re the steward of the child’s growth and weight curve, and if something’s looking wrong, you’ve got to bring that up to the endocrinologist,” Auchus said. Even if lab results appear normal, changes in growth or weight trends may signal the need for treatment adjustments.

The approval of crinecerfont was based on data from 2 randomized, double-blind, placebo-controlled trials involving 285 patients. Pediatric participants saw an 18% reduction in glucocorticoid doses by the study’s end, while maintaining androgen control. Click here for more on the FDA approval, as covered by Contemporary Pediatrics.

Looking ahead, Auchus believes the drug’s success will inspire further therapeutic innovations.

“There’s a lot of movement in this area,” he said. “Other companies are starting to develop alternative targets for treating this disease.”

References

1. Ebert M. FDA approves Crenessity for treatment of classic congenital adrenal hyperplasia. Contemporary Pediatrics. December 13, 2024. Accessed August 14, 2025. https://www.contemporarypediatrics.com/view/fda-approves-crenessity-for-treatment-of-classic-congenital-adrenal-hyperplasia
2. Fitch J. Richard Auchus, MD: Crinecerfont's positive weight-related outcomes in children with CAH. Contemporary Pediatrics. July 31, 2025. Accessed August 14, 2025. https://www.contemporarypediatrics.com/view/richard-auchus-md-crinecerfont-s-positive-weight-related-outcomes-in-children-with-cah