Crinecerfont for Congenital Adrenal Hyperplasia Now Commercially Available, Neurocrine Biosciences Announces

Neurocrine Biosciences announced today that crinecerfont, a first-in class therapy for adults and children with congenital adrenal hyperplasia (CAH), is commercially available in the US.

The novel selective oral corticotropin-releasing factor type 1 receptor (CRF1) antagonist was recently approved by the FDA as an adjunctive treatment to glucocorticoid replacement therapy in adults and children aged 4 years and older with the chronic endocrine disorder.

Crinecerfont directly reduces adrenocorticotropic hormone and downstream adrenal androgen production that characterize the disorder. It is the first treatment for classic CAH that allows people to take lower doses of glucocorticoids while maintaining or improving 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," Kyle W Gano, PhD, Neurocrine chief executive officer, said in the December 20 company announcement. "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."

According to Neurocrine, crinecerfont is exclusively available through specialty pharmacy PANTHERx Rare, which now has CAH-trained pharmacists available 24/7 to answer questions and address concerns of patients, caregivers and health care professionals.

The company is also offering support for the CAH community to obtain crinecerfont through Neurocrine Access Support, a free, comprehensive assistance program designed to ensure patients and all involved with their care are fully prepared to initiate and continue treatment. The company expects 90% of patients will have a monthly copay of $12 or less, according to the announcement, and said that care coordinators are available to assist with navigating the insurance process and identifying other financial assistance options.

The December 13th approval of crinecerfont was supported by the pivotal phase 3 CAHtalyst Pediatric and CAHtalyst Adult clinical trials, the largest-ever clinical trial program of classic CAH.

The enzyme deficiency that characterizes CAH, a rare genetic condition, alters the production of essential adrenal steroid hormones, eg, cortisol, aldosterone and adrenal androgens. Approximately 95% of CAH cases are caused by variants of the CYP21A2 gene that leads to deficiency of the enzyme 21-hydroxylase. Severe 21-OH deficiency leads to suppression of cortisol production and of aldosterone production in approximately three-quarters of affected individuals. 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.

For more information about crinecerfont availability, visit www.nbiaccess.com/crenessity or call 1-855-CRNSITY (276-7489) Monday-Friday 8 am-8 pm ET.

Find more details on CAH and the evolution of pharmacotherapy for the condition in the Patient Care Online CAH Provider Toolkit, including:

CAH management across the lifespan

The CRF pathway interrupted: Potential to Transform CAH Care

Monitoring glucocorticoid therapy in CAH

Treating CAH: Challenges in the transition to adult care

For interviews with the lead investigator for the CAHtalyst Adult trial, see

Expert Perspectives 2024: Breakthrough Therapy for Congenital Adrenal Hyperplasia with PI Richard Auchus, MD, PhD

Source: Neurocrine Biosciences announces commercial availability of CRENESSITY™ (crinecerfont) for children and adults with congenital adrenal hyperplasia. News release. Neurocrine Biosciences. December 20, 2024. Accessed December 20, 2024. https://neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-announces-commercial-availability