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US FDA approves Neurocrine Biosciences’ genetic disorder drug

By Thomson Reuters Dec 13, 2024 | 4:50 PM

(Reuters) -The U.S. Food and Drug Administration has approved Neurocrine Biosciences’ drug to treat a type of genetic disorder, the health regulator’s website showed on Friday.

The drug, branded as Crenessity, is to be used together with glucocorticoid, a type of steroid, to control androgen levels in adults and pediatric patients aged 4 years and older with classic congenital adrenal hyperplasia (CAH).

The drug is expected to be commercially available in about one week, a company spokesperson said.

CAH is a rare genetic disorder affecting the adrenal glands, which produce hormones such as cortisol and androgens.

Patients with the disease do not produce enough cortisol — an essential hormone that regulates stress levels — and produce too many androgens, a testosterone-like hormone.

Crenessity works by reducing excessive production of androgens, resulting in less amount of glucocorticoid treatment needed.

Patients usually require high doses of glucocorticoids, beyond what is required to replace cortisol, because they also help lower excess androgen levels.

(Reporting by Sriparna Roy in Bengaluru; Editing by Alan Barona)