On Friday, the FDA approved Neurocrine Biosciences, Inc.(NASDAQ:NBIX) Crenessity (crinecerfont) capsules and oral solution as an adjunctive treatment to glucocorticoid replacement to control androgens in adult and pediatric patients four years of age and older with classic congenital adrenal hyperplasia (CAH).

CAH is a rare, inherited disorder that affects the adrenal glands and causes them to be larger than normal. The adrenal glands are located above the kidneys and produce hormones that regulate blood sugar, blood pressure, and other bodily functions.

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Crenessity, a potent and selective oral corticotropin-releasing factor type 1 receptor antagonist, is the first and only classic CAH treatment that directly reduces excess adrenocorticotropic hormone and downstream adrenal androgen production, allowing for glucocorticoid dose reduction.

Crenessity is expected to be commercially available in approximately one week. To centralize and simplify prescription fulfillment, the medication will be provided through PANTHERx Rare, a specialty pharmacy.

William Blair sees significant potential in the CAH market but acknowledges that for this to be a blockbuster $1 billion-plus commercial opportunity, there will need to be both patient and clinician education that supraphysiological (amounts greater than normally found in the body) glucocorticoid dosing would no longer be the only way to control androgen levels and prevent adrenal crises.

The analyst writes that focusing on educational initiatives and collaborating with patient advocacy groups, such as the CARES Foundation, is a smart move as Neurocrine prepares for its product launch.

While early-stage competitors like Crinetics Pharmaceuticals, Inc (NASDAQ:CRNX) show promising data, Neurocrine’s position as the first to market in treating congenital adrenal hyperplasia gives it a strong advantage.

Although investors have mixed opinions about the potential success of crinecerfont, William Blair sees opportunities for it to become a blockbuster drug alongside Ingrezza in the long run.

The analyst maintains the Outperform rating on Neurocrine.

Price Action: NBIX stock is up 2.80% at $130.25 premarket at last check Monday.

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This article Neurocrine's Drug Gains FDA Nod As First Targeted Treatment For Certain Type Of Inherited Disorder, Analyst Highlights Blockbuster Potential originally appeared on Benzinga.com

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