Crinecerfont, a CRF1 antagonist, could help reduce glucocorticoid doses for individuals with CAH and improve physical and emotional quality of life, says leading researcher.
Crinecerfont, an investigational corticotropin releasing factor 1 (CRF1) antagonist, was recently granted Priority Review by the US FDA and if approved in December will be the first new treatment for congenital adrenal hyperplasia (CAH) in 70 years. The new drug acts by intercepting hypothalamic signals to the pituitary to trigger release of adrenocorticotropin hormone (ACTH); reduced levels of ACTH lower the stimulus for adrenal production of androgens, which, in excess, cause the myriad metabolic and hormonal aberrations that characterize CAH. The nonglucocorticoid CRF1 antagonist has the potential to significantly reduce glucocorticoid (GC) dosing and the dangerous effects of long-term GC use.
In this short video, Richard Auchus, MD, PhD, principal investigator of the CAHtalyst Adult phase 3 clinical trial of crinecerfont provides insight on how the new drug could help simplify monitoring management of CAH and importantly how the drug's effects may improve individual self care and quality of life.
Richard J Auchus, MD, PhD, is the James A. Shayman and Andrea S. Kevrick professor of translational medicine, professor of internal medicine, and professor of pharmacology at the University of Michigan Medical School in Ann Arbor, MI. His areas of work include molecular and genetic mechanisms of human hypertension, improved diagnostic studies and management in primary aldosteronism and Cushing syndromes, modifier genes in 21-hydroxylase deficiency, cardiovascular disease in polycystic ovary syndrome, and the endocrinology of traumatic brain injury.