"Neurocrine Biosciences, Inc, a leading neuroscience-focused, biopharmaceutical company with a simple purpose to relieve suffering for people with great needs and but few options, has announced CRENESSITY (crinecerfont) is now commercially available in the United States.
Crinecerfont was recently approved by the US Food and Drug Administration 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).
Crinecerfont, a potent and selective oral corticotropin-releasing factor type 1 receptor (CRF1) antagonist, is a first-in-class therapy for classic CAH that directly reduces adrenocorticotropic hormone and downstream adrenal androgen production. It is the first and only classic CAH treatment that allows people to take lower doses of glucocorticoids while maintaining or improving their androgen levels.
“Individuals with CAH and their families have faced ongoing challenges with managing the condition with high-dose steroids alone for the past 70 years,” said Kyle W. Gano, CEO of Neurocrine Biosciences. “We’re proud to now provide crinecerfont to the community, and we are committed to supporting patients in obtaining treatment with crinecerfont through our comprehensive assistance program.”
CRENESSITY is exclusively available through PANTHERx Rare, a specialty pharmacy, to centralize and simplify CRENESSITY prescriptions. PANTHERx Rare has CAH-trained pharmacists available 24/7 to patients, caregivers and healthcare providers to answer questions and address concerns.
Neurocrine Biosciences is committed to supporting patients in obtaining treatment with CRENESSITY by offering Neurocrine Access Support, a free, comprehensive assistance program created for patients, caregivers and healthcare providers. It offers a range of options specifically designed to ensure patients with CAH have everything they need to begin and continue taking CRENESSITY. A dedicated care coordinator, backed by a team, is available to help patients and caregivers navigate the insurance process and identify appropriate financial assistance options.
The US FDA approval of CRENESSITY was supported by the largest-ever clinical trial program of classic CAH, the CAHtalyst Pediatric study, conducted in ages four to 17, and the CAHtalyst Adult study.
CAH is a rare genetic condition that 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% 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% 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.
Historically, exogenous glucocorticoids (GCs) have been used not only to correct the endogenous cortisol deficiency, but doses used are higher than cortisol replacement needed (supraphysiologic) to lower the levels of adrenocorticotropic hormone (ACTH) and adrenal androgens. However, GC treatment at high doses has been associated with serious and significant complications of steroid excess, including metabolic issues such as weight gain and diabetes, cardiovascular disease and osteoporosis. Additionally, long-term treatment with high-dose GCs may have psychological and cognitive impact, such as changes in mood and memory. Adrenal androgen excess has been associated with abnormal bone growth and development in pediatric patients, female health problems such as excess facial hair growth and menstrual irregularities, testicular rest tumors in males and fertility issues in both sexes.
The phase 3 CAHtalyst global registrational studies were designed to evaluate the safety, efficacy and tolerability of CRENESSITY in children and adults with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The CAHtalyst studies were the largest-ever clinical trial program in classic CAH, including 285 pediatric and adult patients.
The CAHtalyst Pediatric study included 103 pediatric patients aged four to 17 years. The study tested two questions. The first question evaluated whether four weeks of CRENESSITY treatment could improve androgen control. The second question evaluated whether an additional 24 weeks of CRENESSITY treatment enabled customized glucocorticoid (GC) down-titration while androstenedione levels were maintained or improved. The CAHtalyst Adult study included 182 adult patients aged 18 to 58 years. Similarly, the first question of the study evaluated whether four weeks of CRENESSITY treatment could improve androgen control, and the second question evaluated whether an additional 20 weeks of CRENESSITY treatment enabled GC reduction to physiologic range while androstenedione levels were maintained or improved.