Neurocrine’s First New CAH Treatment in 70 Years Gets FDA Approval

Neurocrine Biosciences, Inc. has received FDA approval for Crenessity (crinecerfont), marking a major breakthrough for CAH treatment. This first-in-class oral therapy addresses a 70-year treatment gap. CAH is a life-threatening endocrine disorder with no new treatment options for decades. Neurocrine will receive a priority review voucher with the approval, which it could sell for approximately $150 million.

Infertility, Growth Issues, and Adrenal Crisis No Longer an Issue for 30,000 CAH Affected Patients 

Neurocrine Biosciences has introduced a new FDA-approved therapy for classic congenital adrenal hyperplasia (CAH), a rare endocrine disorder traditionally treated with steroids that cause various side effects. The company’s non-steroidal drug offers a potential blockbuster alternative.

CAH is a rare genetic disorder that causes a dangerous imbalance between cortisol and androgen hormones. In people with CAH, the body cannot produce enough cortisol, prompting an increase in adrenocorticotropic hormone (ACTH). This surge in ACTH forces the adrenal glands to overproduce androgens, such as testosterone, which leads to a range of serious and potentially life-threatening symptoms.

The approval of Crenessity marks a critical turning point for individuals living with CAH. An estimated 30,000 people in the U.S. alone are affected by the disorder, facing severe health complications such as infertility, growth issues, and, in some cases, adrenal crisis. Despite its prevalence, treatment options for CAH remained limited for decades. With the recent FDA approval of Crenessity, patients now have access to a groundbreaking therapy.

Kyle W. Gano, Chief Executive Officer of Neurocrine Biosciences, expressed, “The approval of Crenessity is a significant milestone for the CAH community, and we are grateful to the individuals who participated in our clinical trials, including their families and caregivers, and to the clinical investigators who helped advance a new therapy and class of medicines.”

Crenessity Reduces Glucocorticoid Dosage, Controlling the Overproduction of ACTH, & Offering a Safer Treatment 

Traditional treatments for CAH typically involve synthetic glucocorticoids to replace deficient cortisol. However, patients require supraphysiologic doses, exceeding natural production levels, to control the overproduction of ACTH and adrenal androgens effectively. These elevated doses can cause serious side effects, such as weight gain, diabetes, cardiovascular disease, osteoporosis, and psychological or cognitive issues.

Dina Matos, Executive Director of the CARES Foundation, explained, “Patients and families struggle to achieve balance between managing the symptoms of CAH and the side effects or complications of treatment with high-dose steroids, which may impact quality of life.”

Crenessity provides an advanced treatment by targeting the CRF1 receptor, inhibiting ACTH secretion, and reducing adrenal androgen production effectively. This innovative mechanism allows for lower glucocorticoid doses, providing a safer, more effective alternative to long-term steroid use. Moreover, the FDA approved Crenessity based on Phase 3 study results showing it significantly reduces glucocorticoid requirements in CAH patients. The most common side effects—fatigue, headache, dizziness, and muscle or joint pain—were mild to moderate and temporary.

Crenessity is expected to become commercially available to patients within the next week.To streamline the prescription process, PANTHERx Rare, a specialty pharmacy, will distribute the medication and simplify prescription fulfillment for patients.This approval marks a significant milestone in the treatment of CAH, offering new hope for patients struggling with this rare and challenging condition.

Author

Denisse Sandoval