Crinecerfont
Pharmaceutical compound
From Wikipedia, the free encyclopedia
Crinecerfont, sold under the brand name Crenessity, is a medication used for the treatment of congenital adrenal hyperplasia.[1] It is a corticotropin-releasing factor type 1 receptor (CRF1R) antagonist developed to treat classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency (21OHD).[1] It is taken by mouth.[1]
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| Trade names | Crenessity |
| Other names | SSR-125543, NBI-74788 |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a625009 |
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| Routes of administration | By mouth |
| Drug class | Corticotropin-releasing factor type 1 receptor antagonist |
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| Formula | C27H28ClFN2OS |
| Molar mass | 483.04 g·mol−1 |
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The most common side effects of crinecerfont in adults include fatigue, dizziness, and arthralgia (joint pain).[2] For children, the most common side effects include headache, abdominal pain, and fatigue.[2]
Crinecerfont was approved for medical use in the United States in December 2024.[2][3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[4]
Medical uses
Adverse effects
The US prescribing information for crinecerfont has a warning for acute adrenal insufficiency or adrenal crisis.[2]
History
Crinecerfont's approval is based on two randomized, double-blind, placebo-controlled trials in 182 adults and 103 children with classic congenital adrenal hyperplasia.[2] In the first trial, 122 adults received crinecerfont twice daily and 60 received placebo twice daily for 24 weeks.[2] After the first four weeks of the trial, the glucocorticoid dose was reduced to replacement levels, then adjusted based on levels of androstenedione, an androgen hormone.[2] The primary measure of efficacy was the change from baseline in the total glucocorticoid daily dose while maintaining androstenedione control at the end of the trial.[2] The group that received crinecerfont reduced their daily glucocorticoid dose by 27% while maintaining control of androstenedione levels, compared to a 10% daily glucocorticoid dose reduction in the group that received placebo.[2]
In the second trial, 69 children received crinecerfont twice daily and 34 received placebo twice daily for 28 weeks.[2] The primary measure of efficacy was the change from baseline in serum androstenedione at week four.[2] The group that received crinecerfont experienced a statistically significant reduction from baseline in serum androstenedione, compared to an average increase from baseline in the placebo group.[2] At the end of the trial, children assigned to crinecerfont were able to reduce their daily glucocorticoid dose by 18% while maintaining control of androstenedione levels compared to an almost 6% daily glucocorticoid dose increase in children assigned to placebo.[2]
The adult trial was conducted at 54 sites in 16 countries in North America, Europe, and Asia.[5] The pediatric trial was conducted at 37 sites in 10 countries in North America and Europe.[5] Of the 285 participants, 127 (45%) were from the United States.[5] Findings from both clinical trials established crinecerfont safety and efficacy.[5]
The US Food and Drug Administration (FDA) granted the application for crinecerfont fast track, breakthrough therapy, orphan drug, and priority review designations.[2] The FDA granted the approval of Crenessity to Neurocrine Biosciences, Inc.[2]
