Neurocrine Biosciences, Inc., recognized for its neuroscience innovations, has announced the U.S. Food and Drug Administration’s endorsement of Crenessity (crinecerfont) as a supplementary therapy to glucocorticoid replacement. This approval is for the purpose of controlling androgen levels in adults and children aged four and up who suffer from classic congenital adrenal hyperplasia (CAH). CAH is a rare, severe genetic ailment affecting the adrenal glands. As the first direct treatment to curb excess adrenocorticotropic hormone (ACTH) and resultant adrenal androgen production, Crenessity offers the potential to reduce glucocorticoid dosing. This represents a major advancement in the management of classic CAH.
Dr. Kyle W. Gano, CEO of Neurocrine Biosciences, remarked on the landmark approval: “For over 30 years, alongside our late founder, Wylie W. Vale, we have pioneered research that unveiled the significance of corticotropin-releasing factor and its receptor, CRF1, in CAH’s pathophysiology. We extend our gratitude to those who partook in our clinical trials, their families, and the investigators who contributed to ushering in this novel class of medication.”
Dina Matos from CARES Foundation articulated appreciation for Neurocrine Biosciences: “Balancing CAH symptoms against steroid treatment side effects is a challenge influencing life quality. Thanks to Neurocrine Biosciences’ engagement with our community throughout development, Crenessity offers a solution by diminishing both adrenal androgen excess and high-dose steroid requirements.”
Set to become commercially available shortly via PANTHERx Rare—a specialty pharmacy—Crenessity aims to streamline prescription processes. Furthermore, Neurocrine Biosciences is providing free assistance through Neurocrine Access Support to facilitate treatment initiation and continuity. This service includes guidance from a Care Coordinator, helping with insurance navigation and financial assistance options. Most patients will incur costs of $10 or less monthly.
Crenessity’s FDA approval comes off the back of extensive clinical trials—the largest conducted for classic CAH—presenting data in The New England Journal of Medicine that validate its efficacy and safety. Data from the CAHtalyst studies demonstrated improved androgen control in both children and adults.
For children, the CAHtalyst Paediatric study showed significant decreases in androstenedione levels and a reduction in glucocorticoid doses, proving its superiority over placebo treatments. In adults, Crenessity facilitated glucocorticoid dose reduction while maintaining or enhancing androstenedione levels when compared to placebo results.
Despite demonstrating mild and moderate short-term side effects, Crenessity has been tolerated well without severe adverse events emerging from the CAHtalyst studies. Risks such as adrenal insufficiency and crisis remain, demanding careful glucocorticoid management in affected individuals.
Classic CAH stems from a deficiency in the enzyme 21-hydroxylase due to CYP21A2 gene mutations, disrupting adrenal steroid hormone production. Historically managed with high-dose glucocorticoids to normalize hormone levels, challenges associated with their extended use, such as metabolic and psychological complications, have highlighted the need for alternatives.
Crenessity, a CRF1 receptor antagonist, provides an innovative non-glucocorticoid pathway to control ACTH and adrenal androgens. It’s offered as 50 mg and 100 mg capsules or as a 50 mg/mL oral solution. Proper dosing, integrated into ongoing glucocorticoid therapy, requires healthcare providers’ oversight to ensure tailored patient care.