CRENESSITY offers a novel approach to managing classic congenital adrenal hyperplasia (CAH) in adults and children aged 4 years and older. This medication is designed to work alongside glucocorticoid replacement therapy to help control androgen levels and improve treatment outcomes. CRENESSITY was approved on December 13, 2024.
What is the name of the drug, and what does it do?
CRENESSITY (pronounced kreh-NEH-sih-tee) contains crinecerfont, which is a corticotropin-releasing factor type 1 receptor antagonist. It helps regulate androgen (testosterone-like hormone) levels, which are often elevated in people with CAH. The drug is used as an add-on treatment to glucocorticoids to enhance androgen control and potentially reduce glucocorticoid dosage over time.
How does it work?
CRENESSITY works by blocking corticotropin-releasing factor (CRF) type 1 receptors in the pituitary gland. This reduces the release of adrenocorticotropic hormone (ACTH), which, in turn, lowers adrenal androgen production. By targeting this specific pathway, the drug helps control hormone levels while supporting adrenal gland function.
What did the research discover?
In clinical trials involving both adults and children with CAH, CRENESSITY demonstrated significant benefits. Among adults, a 27% reduction in glucocorticoid doses was achieved, while maintaining stable androgen levels, compared to a 10% reduction in the placebo group. In children, the drug led to improved androgen control, with serum androstenedione levels significantly reduced compared to placebo. These findings highlight CRENESSITY’s ability to enhance treatment outcomes while potentially reducing the long-term side effects associated with high doses of glucocorticoids.
What are some of the side effects?
The most common side effects of CRENESSITY include:
- In adults: Fatigue, headache, dizziness, joint pain, back pain, decreased appetite, and muscle pain
- In children: Headache, abdominal pain, nasal congestion, nosebleeds, and fatigue
Rare but serious side effects include hypersensitivity reactions and the risk of adrenal insufficiency if glucocorticoid therapy is not adequately managed.
What are the dosage recommendations, and how is it prescribed?
CRENESSITY is taken orally twice daily with meals. The dosage for adults is 100 mg in the morning and evening, while pediatric dosages are weight-based:
- 25 mg twice daily for children weighing 10–20 kg
- 50 mg twice daily for children weighing 20–55 kg
- 100 mg twice daily for children weighing over 55 kg
Patients must continue glucocorticoid therapy during treatment with CRENESSITY and adjust doses only under medical supervision.
Source
Neurocrine Biosciences, Inc. Prescribing information for CRENESSITY (crinecerfont). December 2024. Available at:https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/218808s000,218820s000lbl.pdf
