FDA Approves Neurocrine Biosciences’ Therapy for Rare Endocrine Disorder

Rare Daily Staff

The U.S. Food and Drug Administration approved Neurocrine Biosciences Crenessity to treat children and adults with the rare endocrine disorder classic congenital adrenal hyperplasia, the first new therapy for the condition in 70 years.

The agency approved Crenessity 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).

Congenital adrenal hyperplasia (CAH)  is a rare, serious, and lifelong genetic condition involving the adrenal glands. It results in an enzyme deficiency that alters the production of adrenal steroid hormones, such as cortisol, aldosterone and adrenal androgens, which are essential for life. Approximately 95 percent of CAH cases are caused by variants of the CYP21A2 gene that leads to deficiency of the enzyme 21-hydroxylase (21-OH). Severe deficiency of this enzyme leads to an inability of the adrenal glands to produce enough cortisol and, in approximately 75 percent of cases, aldosterone.

Because individuals with CAH are still able to produce androgens, the unused precursors that would normally be used to make cortisol instead result in the production of excess amounts of androgens. If left untreated, CAH can result in salt wasting, dehydration, and even death.

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

The FDA approval is supported by the largest-ever clinical trial program of classic CAH, the CAHtalyst Pediatric and Adult Phase 3 global registrational studies. CAHtalyst phase 3 data results in pediatric and adult patients with classic CAH were published in the New England Journal of Medicine.

In both CAHtalyst studies, Crenessity enabled lower steroid doses and decreased androgen levels.

Crenessity was well tolerated with few treatment-related adverse events in both CAHtalyst studies. Pediatric and adult patients taking Crenessity had no treatment-related serious adverse events.

“For the last three decades, Neurocrine Biosciences, together with our late founder, Wylie W. Vale, has conducted groundbreaking research uncovering the critical role of corticotropin-releasing factor and its receptor, CRF1, in the pathophysiology of congenital adrenal hyperplasia,” said Kyle Gano, CEO of Neurocrine Biosciences. “The approval of Crenessity is a significant milestone for the CAH community.”