Pharma Focus Asia

Neurocrine Biosciences Introduces Novel Crinecerfont for Congenital Adrenal Hyperplasia

Neurocrine Biosciences has introduced crinecerfont as a novel treatment for congenital adrenal hyperplasia (CAH).

Crinecerfont is an investigational oral medication designed as a selective corticotropin-releasing factor type 1 receptor (CRF1) antagonist. It is being developed with the aim of managing and reducing excess adrenal androgens in congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21-OHD), operating through a mechanism independent of steroid use. 

By antagonising CRF1 receptors in the pituitary, it has demonstrated the capability to lower adrenocorticotropic hormone (ACTH) levels. This, in turn, may lead to a decrease in adrenal androgen production, potentially alleviating the symptoms associated with classic CAH.

Congenital adrenal hyperplasia (CAH) represents a cluster of genetic conditions triggering enzyme deficiencies that disrupt the production of critical adrenal hormones crucial for life. About 95 percent of CAH cases stem from a mutation causing 21-hydroxylase (21-OHD) deficiency.

In classic CAH, a severe deficit in this enzyme impedes the adrenal glands' ability to generate cortisol and, in roughly 75 percent of instances, aldosterone. Without treatment, this deficiency can lead to salt wasting, dehydration, and potentially fatal consequences.

Currently, classic CAH doesn't have approved treatments that don't involve glucocorticoids. Glucocorticoids are the standard care, utilised not only to address cortisol deficiency but often administered at higher-than-normal doses (supraphysiologic) to help lower the elevated levels of corticotropin-releasing factor (CRF) and adrenocorticotropic hormone (ACTH), which contribute to excess androgen production.

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