Neurocrine Biosciences Announces Publication on Traditional Glucocorticoid Treatment in Classic Congenital Adrenal Hyperplasia in Expert Review of Endocrinology & Metabolism
Neurocrine Biosciences (NBIX) announced the publication of a narrative review discussing challenges in treating classic congenital adrenal hyperplasia (CAH) with traditional glucocorticoids (GCs) and potential benefits of new non-GC treatments. The review, published in Expert Review of Endocrinology & Metabolism, highlights how non-GC mechanisms could enable lower, more physiologic GC dosing while managing excess ACTH and androgens.
The publication emphasizes that chronic exposure to high-dose GC treatment can lead to severe cardiovascular, metabolic, and skeletal complications, affecting patients' mental health and quality of life. The FDA recently approved CRENESSITY™ (crinecerfont), the first and only classic CAH treatment that directly reduces excess ACTH and adrenal androgen production, allowing for GC dose reduction in patients four years and older.
Neurocrine Biosciences (NBIX) ha annunciato la pubblicazione di una revisione narrativa che discute le sfide nel trattamento dell'iperplasia surrenalica congenita classica (CAH) con glucocorticoidi tradizionali (GC) e i potenziali benefici di nuovi trattamenti non-GC. La revisione, pubblicata su Expert Review of Endocrinology & Metabolism, sottolinea come i meccanismi non-GC potrebbero consentire dosaggi di GC più bassi e fisiologici, gestendo al contempo l'eccesso di ACTH e androgeni.
La pubblicazione evidenzia che l'esposizione cronica a trattamenti con alte dosi di GC può portare a gravi complicazioni cardiovascolari, metaboliche e scheletriche, influenzando la salute mentale e la qualità della vita dei pazienti. Recentemente, la FDA ha approvato CRENESSITY™ (crinecerfont), il primo e unico trattamento per la CAH classica che riduce direttamente l'eccesso di ACTH e la produzione di androgeni surrenalici, consentendo una riduzione della dose di GC nei pazienti di quattro anni e oltre.
Neurocrine Biosciences (NBIX) anunció la publicación de una revisión narrativa que discute los desafíos en el tratamiento de la hiperplasia suprarrenal congénita clásica (CAH) con glucocorticoides tradicionales (GC) y los beneficios potenciales de nuevos tratamientos no-GC. La revisión, publicada en Expert Review of Endocrinology & Metabolism, destaca cómo los mecanismos no-GC podrían permitir dosis de GC más bajas y fisiológicas, al mismo tiempo que se manejan el exceso de ACTH y andrógenos.
La publicación enfatiza que la exposición crónica a tratamientos con dosis altas de GC puede llevar a graves complicaciones cardiovasculares, metabólicas y esqueléticas, afectando la salud mental y la calidad de vida de los pacientes. Recientemente, la FDA aprobó CRENESSITY™ (crinecerfont), el primer y único tratamiento para la CAH clásica que reduce directamente el exceso de ACTH y la producción de andrógenos suprarrenales, permitiendo una reducción de la dosis de GC en pacientes de cuatro años en adelante.
Neurocrine Biosciences (NBIX)는 전통적인 글루코코르티코이드(GC)로 고전적인 선천성 부신hyperplasia(CAH)의 치료에 대한 도전과 새로운 비-GC 치료의 잠재적인 이점을 다룬 내러티브 리뷰의 발표를 알렸습니다. 이 리뷰는 Expert Review of Endocrinology & Metabolism에 게재되었으며, 비-GC 메커니즘이 ACTH 및 안드로겐 과다를 관리하면서 더 낮고 생리적인 GC 용량을 가능하게 할 수 있음을 강조합니다.
출판물은 고용량 GC 치료에 대한 만성 노출이 환자의 정신 건강과 삶의 질에 영향을 미치는 심각한 심혈관, 대사 및 골격 합병증으로 이어질 수 있음을 강조합니다. FDA는 최근 CRENESSITY™ (crinecerfont)를 승인했으며, 이는 ACTH 및 부신 안드로겐 생산을 직접적으로 줄이는 최초이자 유일한 고전 CAH 치료제로, 4세 이상의 환자에서 GC 용량 감소를 가능하게 합니다.
Neurocrine Biosciences (NBIX) a annoncé la publication d'une revue narrative discutant des défis dans le traitement de l'hyperplasie surrénalienne congénitale classique (CAH) avec des glucocorticoïdes traditionnels (GC) et des bénéfices potentiels de nouveaux traitements non-GC. La revue, publiée dans l'Expert Review of Endocrinology & Metabolism, souligne comment les mécanismes non-GC pourraient permettre de réduire les doses de GC à des niveaux plus bas et physiologiques tout en gérant les excès d'ACTH et d'androgènes.
La publication souligne que l'exposition chronique à un traitement par GC à forte dose peut entraîner de graves complications cardiovasculaires, métaboliques et squelettiques, affectant la santé mentale et la qualité de vie des patients. La FDA a récemment approuvé CRENESSITY™ (crinecerfont), le premier et unique traitement de la CAH classique qui réduit directement l'excès d'ACTH et la production d'androgènes surrénaliens, permettant ainsi une réduction de la dose de GC chez les patients âgés de quatre ans et plus.
Neurocrine Biosciences (NBIX) gab die Veröffentlichung eines narrativen Reviews bekannt, der sich mit den Herausforderungen bei der Behandlung der klassischen kongenitalen adrenalen Hyperplasie (CAH) mit traditionellen Glukokortikoiden (GC) sowie den potenziellen Vorteilen neuer nicht-GC-Behandlungen befasst. Der Review, veröffentlicht in der Expert Review of Endocrinology & Metabolism, hebt hervor, wie nicht-GC-Mechanismen niedrigere, physiologische GC-Dosierungen ermöglichen könnten, während gleichzeitig der Überschuss an ACTH und Androgenen gemanagt wird.
Die Publikation betont, dass die chronische Exposition gegenüber hochdosierter GC-Behandlung zu schwerwiegenden kardiovaskulären, metabolischen und skeletalen Komplikationen führen kann, die die psychische Gesundheit und die Lebensqualität der Patienten beeinträchtigen. Die FDA hat kürzlich CRENESSITY™ (crinecerfont) genehmigt, die erste und einzige Behandlung der klassischen CAH, die direkt überschüssiges ACTH und die Produktion von adrenal Androgenen reduziert und eine Reduzierung der GC-Dosis bei Patienten ab vier Jahren ermöglicht.
- FDA approval of CRENESSITY as first-in-class treatment for CAH
- Product addresses significant unmet medical need in CAH treatment
- Potential for reduced complications through lower glucocorticoid dosing
- None.
Insights
The publication of this narrative review in Expert Review of Endocrinology & Metabolism represents a critical milestone in understanding the treatment evolution for classic CAH. The core advancement centers on CRENESSITY, which operates through a novel CRF1 receptor antagonist mechanism, fundamentally different from traditional glucocorticoid therapy.
The therapeutic significance lies in its ability to reduce ACTH and androgen production without relying on high-dose glucocorticoids. Think of it as adding a precision tool to a toolbox that previously contained only a sledgehammer - while effective, the old approach often caused collateral damage. This new treatment paradigm could revolutionize CAH management by allowing for lower glucocorticoid doses while maintaining hormonal control.
The Mayo Clinic's involvement and endorsement through Dr. Bancos adds substantial credibility to these findings. The emphasis on lifetime cumulative benefits of reduced glucocorticoid exposure represents a important shift in treatment philosophy, moving from acute symptom management to long-term health optimization.
This development positions Neurocrine Biosciences favorably in the rare disease market. With CRENESSITY's FDA approval and this supporting publication, the company has established a strong foothold in the CAH treatment space. The market opportunity is significant - while CAH is rare, the chronic nature of the condition and the current limitations of traditional treatments create a compelling value proposition.
The timing of this publication is strategic, following the FDA approval, as it helps build credibility and awareness among healthcare providers. This educational approach typically translates to faster adoption rates and better market penetration. For investors, this represents a potential growth catalyst as CRENESSITY could become the new standard of care in CAH treatment, particularly given its first-mover advantage and unique mechanism of action.
- Narrative Review Assesses Current Treatment Challenges and the Evolving Classic Congenital Adrenal Hyperplasia (CAH) Therapeutic Landscape
- Non-Glucocorticoid (GC) Mechanisms for Treatment of CAH Could Enable Control of Excess ACTH and Androgens Without the Need for High-Dose GCs, Reducing Related Complications Over a Lifetime
Treatment of classic CAH is a lifelong challenge that involves balancing the need to manage excess adrenocorticotropic hormone (ACTH) and androgens — historically achieved through high doses of GCs — while managing the risks associated with GC-related complications. The review, Glucocorticoid Therapy in Classic Congenital Adrenal Hyperplasia: Traditional and New Treatment Paradigms, provides information for healthcare providers as they navigate the evolving classic CAH therapeutic landscape.
"The treatment paradigm for CAH involves continually monitoring and balancing ACTH and androgen levels and glucocorticoid-dosing to optimize treatment," said Irina Bancos, M.S., M.D., Professor of Medicine, Division of Endocrinology at the Mayo Clinic. "New CAH medications that control excess ACTH and adrenal androgens through a non-glucocorticoid mechanism allow for glucocorticoid dose reduction, with the cumulative reduction over time translating into a significant decrease in the risk for complications that can occur when higher doses are used over a patient's lifetime."
"Chronic exposure to high-dose glucocorticoid treatment can result in severe cardiovascular, metabolic and skeletal complications, as well as negatively impact the mental health and quality of life of patients," said Eiry W. Roberts, M.D., Chief Medical Officer, Neurocrine Biosciences. "Over a lifetime, even modest reductions in daily glucocorticoid doses can reduce the risk of these complications and lessen the overall burden of glucocorticoid exposure."
CAH presents a significant challenge for individuals living with the condition, their families and the healthcare providers who treat them. The condition leads to excess androgen production and has traditionally required high-dose GC treatment, both of which can have serious consequences. Until recently, treatment options have been limited, highlighting the unmet need for innovative new therapies for CAH.
The FDA recently approved CRENESSITY™ (crinecerfont) as an adjunctive treatment to GC replacement to control androgens in adult and pediatric patients four years of age and older with classic CAH. CRENESSITY, a potent and selective oral corticotropin-releasing factor type 1 receptor (CRF1) antagonist, is the first and only classic CAH treatment that directly reduces excess ACTH and downstream adrenal androgen production, allowing for GC dose reduction.
About Congenital Adrenal Hyperplasia
Congenital adrenal hyperplasia (CAH) is a rare genetic condition that results in an enzyme deficiency that alters the production of adrenal steroid hormones, such as cortisol, aldosterone and adrenal androgens, which are essential for life. Approximately
Historically, exogenous glucocorticoids (GCs) have been used not only to correct the endogenous cortisol deficiency, but doses used are higher than cortisol replacement needed (supraphysiologic) to lower the levels of adrenocorticotropic hormone (ACTH) and adrenal androgens. However, GC treatment at high doses has been associated with serious and significant complications of steroid excess, including metabolic issues such as weight gain and diabetes, cardiovascular disease and osteoporosis. Additionally, long-term treatment with high-dose GCs may have psychological and cognitive impact, such as changes in mood and memory. Adrenal androgen excess has been associated with abnormal bone growth and development in pediatric patients, female health problems such as excess facial hair growth and menstrual irregularities, testicular rest tumors in males and fertility issues in both sexes.
About CRENESSITY™ (crinecerfont)
CRENESSITY™ is a potent and selective, oral corticotropin-releasing factor type 1 receptor (CRF1) antagonist developed to reduce and control excess adrenocorticotropic hormone (ACTH) and adrenal androgens through a non-glucocorticoid (GC) mechanism for the treatment of classic congenital adrenal hyperplasia (CAH). Antagonism of CRF1 receptors in the pituitary has been shown to decrease ACTH levels, which in turn decreases the production of adrenal androgens and potentially the symptoms associated with CAH. The robust clinical study data demonstrate that lowering adrenal androgen levels with CRENESSITY enables lower, more physiologic dosing of GCs to replace missing cortisol.
CRENESSITY comes in capsules and an oral solution. The capsule formulation is available in 50 mg and 100 mg doses. The oral solution is available as a 50 mg/mL strength formulation. For adults 18 years and older, the recommended dosage is 100 mg twice daily taken orally with a meal. For pediatric patients four to 17 years of age weighing less than 55 kg (121 lbs), the recommended dosage is based on body weight and is administered twice daily, taken orally with a meal. For pediatric patients weighing more than 55 kg (121 lbs), the recommended dosage is 100 mg twice daily taken orally with a meal. Healthcare providers can work with patients to determine the appropriate formulation for use depending on patient needs. Patients receiving CRENESSITY should continue GC therapy for cortisol replacement.
Important Information
Approved Uses
CRENESSITY (crinecerfont) is a prescription medicine used together with glucocorticoids (steroids) to control androgen (testosterone-like hormone) levels in adults and children 4 years of age and older with classic congenital adrenal hyperplasia (CAH).
IMPORTANT SAFETY INFORMATION
Do not take CRENESSITY if you:
Are allergic to crinecerfont, or any of the ingredients in CRENESSITY.
CRENESSITY may cause serious side effects, including:
Allergic Reactions. Symptoms of an allergic reaction include tightness of the throat, trouble breathing or swallowing, swelling of the lips, tongue, or face, and rash. If you have an allergic reaction to CRENESSITY, get emergency medical help right away and stop taking CRENESSITY.
Risk of Sudden Adrenal Insufficiency or Adrenal Crisis With Too Little Glucocorticoid (Steroid) Medicine. Sudden adrenal insufficiency or adrenal crisis can happen in people with congenital adrenal hyperplasia who are not taking enough glucocorticoid (steroid) medicine. You should continue taking your glucocorticoid (steroid) medicine during treatment with CRENESSITY. Certain conditions such as infection, severe injury, or shock may increase your risk for sudden adrenal insufficiency or adrenal crisis. Tell your healthcare provider if you get a severe injury, infection, illness, or have planned surgery during treatment. Your healthcare provider may need to change your dose of glucocorticoid (steroid) medicine.
Before taking CRENESSITY, tell your healthcare provider about all of your medical conditions, including if you are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed.
Tell your healthcare provider about all the medicines you take, including prescription and over-the- counter medicines, vitamins, and herbal supplements.
The most common side effects of CRENESSITY in adults include tiredness, headache, dizziness, joint pain, back pain, decreased appetite, and muscle pain.
The most common side effects of CRENESSITY in children include headache, stomach pain, tiredness, nasal congestion, and nose bleeds.
These are not all the possible side effects of CRENESSITY. Call your healthcare provider for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch at www.fda.gov/medwatch or call 1-800-FDA-1088.
Dosage Forms and Strengths: CRENESSITY is available in 50 mg and 100 mg capsules and as an oral solution of 50 mg/mL.
Please see full Prescribing Information
About Neurocrine Biosciences, Inc.
Neurocrine Biosciences is a leading neuroscience-focused, biopharmaceutical company with a simple purpose: to relieve suffering for people with great needs. We are dedicated to discovering and developing life-changing treatments for patients with under-addressed neurological, neuroendocrine and neuropsychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington's disease, classic congenital adrenal hyperplasia, endometriosis* and uterine fibroids,* as well as a robust pipeline including multiple compounds in mid- to late-phase clinical development across our core therapeutic areas. For three decades, we have applied our unique insight into neuroscience and the interconnections between brain and body systems to treat complex conditions. We relentlessly pursue medicines to ease the burden of debilitating diseases and disorders, because you deserve brave science. For more information, visit neurocrine.com, and follow the company on LinkedIn, X (formerly Twitter) and Facebook. (*in collaboration with AbbVie)
The NEUROCRINE BIOSCIENCES Logo Lockup and YOU DESERVE BRAVE SCIENCE are registered trademarks of Neurocrine Biosciences, Inc. CRENESSITY and CAHtalyst are trademarks of Neurocrine Biosciences, Inc.
Forward-Looking Statements
In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. These statements include, but are not limited to, statements regarding the potential benefits to be derived from CRENESSITY for the treatment of classic congenital adrenal hyperplasia (CAH); the value and benefits CRENESSITY brings to patients with CAH; the ability of Neurocrine Biosciences to ensure patients have access to CRENESSITY; and whether the results from our clinical trials of CRENESSITY are indicative of real-world results. Factors that could cause actual results to differ materially from those stated or implied in the forward-looking statements include, but are not limited to, the following: risks and uncertainties associated with Neurocrine Biosciences' business and finances in general, as well as risks and uncertainties associated with the commercialization of CRENESSITY; whether CRENESSITY receives adequate reimbursement from third-party payors; the degree and pace of market uptake of CRENESSITY; risks and uncertainties relating to competitive products and technological changes that may limit demand for CRENESSITY; risks associated with the Company's dependence on third parties for development and manufacturing activities related to CRENESSITY, and the ability of the Company to manage these third parties; risks that additional regulatory submissions for CRENESSITY or other product candidates may not occur or be submitted in a timely manner; risks that the FDA or other regulatory authorities may make adverse decisions regarding CRENESSITY; risks that post-approval CRENESSITY commitments or requirements may be delayed; risks that CRENESSITY may be precluded from commercialization by the proprietary or regulatory rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; risks and uncertainties relating to competitive products and technological changes that may limit demand for CRENESSITY; and other risks described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's quarterly report on Form 10-Q for the quarter ended September 30, 2024. Neurocrine Biosciences disclaims any obligation to update the statements contained in this press release after the date hereof other than required by law.
© 2025 Neurocrine Biosciences, Inc. All Rights Reserved. CAP-CFT-US-0016 01/2025
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FAQ
What is CRENESSITY and how does it treat CAH patients differently from traditional treatments?
What are the main complications of traditional glucocorticoid treatment for CAH that NBIX's CRENESSITY aims to address?
What age groups are eligible for NBIX's CRENESSITY treatment for CAH?
How does NBIX's CRENESSITY work as a CRF1 antagonist for CAH treatment?
