Chemistry:Crinecerfont

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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]

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

Crinecerfont is indicated as adjunctive treatment to glucocorticoid replacement to control androgens in people aged four years of age and older with classic congenital adrenal hyperplasia.[1][2]

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]

Society and culture

Crinecerfont was approved for medical use in the United States in December 2024.[1][2][6]

Names

Crinecerfont is the international nonproprietary name.[7]

Crinecerfont is sold under the brand name Crenessity.[1]

References

  1. ↑ 1.0 1.1 1.2 1.3 1.4 1.5 Cite error: Invalid <ref> tag; no text was provided for refs named Crenessity FDA label
  2. ↑ 2.00 2.01 2.02 2.03 2.04 2.05 2.06 2.07 2.08 2.09 2.10 2.11 2.12 2.13 2.14 2.15 2.16 "FDA Approves New Treatment for Congenital Adrenal Hyperplasia". U.S. Food and Drug Administration (FDA) (Press release). 1 October 2024. Archived from the original on 13 December 2024. Retrieved 16 December 2024. Public Domain This article incorporates text from this source, which is in the public domain.
  3. ↑ "Novel Drug Approvals for 2024". 1 October 2024. https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2024. 
  4. ↑ (PDF) New Drug Therapy Approvals 2024 (Report). January 2025. https://www.fda.gov/media/184967/download. Retrieved 21 January 2025. 
  5. ↑ 5.0 5.1 5.2 5.3 "Drug Trials Snapshot: Crenessity". 13 December 2024. https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshot-crenessity.  Public Domain This article incorporates text from this source, which is in the public domain.
  6. ↑ "Neurocrine Biosciences Announces FDA Approval of Crenessity (crinecerfont), a First-in-Class Treatment for Children and Adults With Classic Congenital Adrenal Hyperplasia" (Press release). Neurocrine Biosciences. 13 December 2024. Retrieved 16 December 2024 – via PR Newswire.
  7. ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 82". WHO Drug Information 33 (3). 2019. 

Further reading



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