Neurocrine Biosciences Announces Classic Congenital Adrenal Hyperplasia Supplement Published Today in The Journal of Clinical Endocrinology & Metabolism
Neurocrine Biosciences (NBIX) announced the publication of a supplement focused on classic congenital adrenal hyperplasia (CAH) in The Journal of Clinical Endocrinology & Metabolism. The supplement, comprising eight review articles, provides comprehensive insights into the challenges and management of classic CAH due to 21-hydroxylase deficiency.
The publication features contributions from leading endocrinologists and researchers, covering topics such as pathophysiology, clinical manifestations, treatment challenges, and advances in treatment, including CRENESSITY™ (crinecerfont), the first oral treatment that directly reduces excess adrenocorticotropic hormone and adrenal androgen production.
The supplement aims to enhance healthcare professionals' understanding of CAH management and improve patient care through evidence-based approaches. It addresses various aspects including genetics, treatment challenges in different age groups, mental health issues, and barriers to management.
Neurocrine Biosciences (NBIX) ha annunciato la pubblicazione di un supplemento focalizzato sulla forma classica di iperplastia surrenale congenita (CAH) nel The Journal of Clinical Endocrinology & Metabolism. Il supplemento, composto da otto articoli di revisione, offre approfondimenti completi sulle sfide e sulla gestione della CAH classica causata da carenza di 21-idrossilasi.
La pubblicazione presenta contributi di eminenti endocrinologi e ricercatori, affrontando temi come la fisiopatologia, le manifestazioni cliniche, le sfide terapeutiche e i progressi nel trattamento, tra cui CRENESSITY™ (crinecerfont), il primo trattamento orale che riduce direttamente la produzione eccessiva di ormone adrenocorticotropo e androgeni surrenali.
Il supplemento mira a migliorare la comprensione da parte dei professionisti sanitari della gestione della CAH e a migliorare le cure per i pazienti attraverso approcci basati su prove. Affronta vari aspetti tra cui genetica, difficoltà nel trattamento in diverse fasce d'età, problemi di salute mentale e ostacoli alla gestione.
Neurocrine Biosciences (NBIX) anunció la publicación de un suplemento enfocado en la hiperplasia suprarrenal congénita clásica (CAH) en The Journal of Clinical Endocrinology & Metabolism. El suplemento, que consta de ocho artículos de revisión, proporciona una visión integral de los desafíos y la gestión de la CAH clásica debida a la deficiencia de 21-hidroxilasa.
La publicación cuenta con contribuciones de destacados endocrinólogos e investigadores, cubriendo temas como fisiopatología, manifestaciones clínicas, desafíos en el tratamiento y avances en la terapia, incluido CRENESSITY™ (crinecerfont), el primer tratamiento oral que reduce directamente la producción excesiva de hormona adrenocorticotrópica y andrógenos suprarrenales.
El suplemento tiene como objetivo mejorar la comprensión de los profesionales de la salud sobre la gestión de la CAH y mejorar la atención al paciente a través de enfoques basados en la evidencia. Aborda diversos aspectos, incluidos la genética, los desafíos en el tratamiento en diferentes grupos de edad, problemas de salud mental y barreras para la gestión.
Neurocrine Biosciences (NBIX)는 The Journal of Clinical Endocrinology & Metabolism에 고전적 선천적 부신비대증(CAH)에 대한 초점을 맞춘 보충 자료를 발표했다고 발표했습니다. 총 8개의 리뷰 기사를 포함하고 있는 이 보충 자료는 21-하이드록실화 효소 결핍으로 인한 고전적 CAH의 관리 및 도전 과제를 포괄적으로 조망합니다.
출판물에는 저명한 내분비학자와 연구자들이 기여하였으며, 병리생리학, 임상 증상, 치료의 어려움 및 CRENESSITY™ (crinecerfont)를 포함한 치료 발전과 같은 주제를 다루고 있습니다. CRENESSITY™는 과도한 부신피질자극호르몬과 부신 안드로겐 생성을 직접 감소시키는 첫 번째 경구 치료제입니다.
이 보충 자료는 의료 전문가들이 CAH 관리에 대한 이해를 높이고 증거 기반 접근 방식을 통해 환자 치료를 개선하는 것을 목표로 합니다. 유전학, 연령대별 치료의 어려움, 정신 건강 문제 및 관리에 대한 장벽 등을 포함한 다양한 측면을 다룹니다.
Neurocrine Biosciences (NBIX) a annoncé la publication d'un supplément axé sur l'hyperplasie surrénalienne congénitale classique (CAH) dans The Journal of Clinical Endocrinology & Metabolism. Le supplément, comprenant huit articles de revue, offre des perspectives complètes sur les défis et la gestion de la CAH classique due à un déficit en 21-hydroxylase.
La publication présente des contributions d'éminents endocrinologues et chercheurs, couvrant des sujets tels que la physiopathologie, les manifestations cliniques, les défis du traitement et les avancées thérapeutiques, notamment CRENESSITY™ (crinecerfont), le premier traitement oral qui réduit directement la production excessive d'hormone adrénocorticotrope et des androgènes surrénaliens.
Le supplément vise à améliorer la compréhension des professionnels de santé quant à la gestion de la CAH et à améliorer les soins aux patients grâce à des approches basées sur des données probantes. Il aborde divers aspects, y compris la génétique, les défis de traitement selon les groupes d'âge, les problèmes de santé mentale et les obstacles à la gestion.
Neurocrine Biosciences (NBIX) hat die Veröffentlichung eines Supplements angekündigt, das sich auf die klassische kongenitale adrenale Hyperplasie (CAH) im The Journal of Clinical Endocrinology & Metabolism konzentriert. Das Supplement, das aus acht Übersichtsartikeln besteht, bietet umfassende Einblicke in die Herausforderungen und das Management der klassischen CAH aufgrund eines 21-Hydroxylasemangels.
Die Veröffentlichung enthält Beiträge von führenden Endokrinologen und Forschern, die Themen wie Pathophysiologie, klinische Manifestationen, Behandlungsschwierigkeiten und Fortschritte in der Therapie, einschließlich CRENESSITY™ (crinecerfont), der ersten oralen Behandlung, die direkt die übermäßige Produktion von adrenocorticotropem Hormon und adrenalem Androgen reduziert, abdecken.
Das Supplement zielt darauf ab, das Verständnis der Gesundheitsdienstleister für das Management von CAH zu verbessern und die Patientenversorgung durch evidenzbasierte Ansätze zu optimieren. Es behandelt verschiedene Aspekte wie Genetik, Behandlungschallenges in unterschiedlichen Altersgruppen, psychische Gesundheitsprobleme und Hindernisse im Management.
- First-to-market position with CRENESSITY for CAH treatment
- Publication in a leading medical journal enhancing scientific credibility
- None.
Insights
The publication of this comprehensive CAH-focused supplement in The Journal of Clinical Endocrinology & Metabolism represents a strategic milestone for Neurocrine Biosciences, particularly in establishing CRENESSITY's position in the CAH treatment landscape. This isn't just another academic publication - it's a carefully orchestrated move to build scientific credibility and market presence.
The supplement's timing is particularly strategic, following CRENESSITY's recent approval as the first non-glucocorticoid treatment for CAH. By featuring eight comprehensive reviews authored by leading endocrinologists, Neurocrine is effectively creating a scientific foundation that could accelerate clinical adoption. The publication serves multiple business objectives:
- Market Education: By comprehensively addressing CAH management challenges, the supplement helps create awareness of the limitations of current glucocorticoid treatments, potentially priming the market for CRENESSITY adoption.
- Scientific Legitimacy: Publication in JCEM, a highly respected journal, provides important scientific validation for Neurocrine's approach to CAH treatment.
- Market Access Strategy: The detailed coverage of treatment barriers and unmet needs could support reimbursement discussions with payers.
From an investor perspective, this publication signifies Neurocrine's commitment to establishing CRENESSITY as the standard of care in CAH treatment. The company's focus on educational initiatives suggests a well-planned commercialization strategy that could drive adoption in this rare disease market, where CRENESSITY faces competition.
For context, the global CAH treatment market represents a significant opportunity, with approximately 30,000 patients in the U.S. alone. By positioning CRENESSITY as the first targeted therapy that allows for glucocorticoid dose reduction, Neurocrine could capture a substantial share of this market, potentially translating to meaningful revenue growth in their rare disease portfolio.
"Our understanding of the genetics, pathophysiology, and complications of CAH has exploded over the last 20 years," said Dr. Richard Auchus, M.D., Ph.D., Principal Investigator, Professor of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes at the University of Michigan. "These reviews capture the many dimensions of this complex condition and its management. The limitations of traditional glucocorticoid treatment, particularly in under-resourced areas, create a large disease burden, multiple co-morbidities, and poor outcomes for many patients. The authors are optimistic that this evolving knowledge and emerging treatments, such as corticotropin-releasing factor type 1 receptor antagonists, hold great promise for more personalized care, with improved androgen control and less glucocorticoid-related health consequences in patients."
The JCEM supplement is a collection of reviews authored by leading endocrinologists and researchers and sponsored by Neurocrine Biosciences. It explores various aspects of classic CAH management, including pathophysiology, clinical manifestations, treatment challenges, barriers to care, psychosocial impact and advances in treatment, including CRENESSITY™ (crinecerfont), a potent and selective oral corticotropin-releasing factor type 1 receptor (CRF1) antagonist. CRENESSITY is the first and only classic CAH treatment that directly reduces excess adrenocorticotropic hormone and downstream adrenal androgen production, allowing for glucocorticoid dose reduction. The articles aim to provide healthcare professionals with up-to-date insights and evidence-based approaches that can enhance clinical practice and patient care.
"We are honored to support this important educational content in collaboration with The Journal of Clinical Endocrinology & Metabolism," said Eiry W. Roberts, M.D., Chief Medical Officer, Neurocrine Biosciences. "Neurocrine is committed to empowering patients, caregivers and healthcare providers with the knowledge and support to navigate the life-long challenges of treating and living with CAH."
The eight reviews include:
- Genetics and Pathophysiology of Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency (Yang M and White PC)
- Clinical Manifestations and Treatment Challenges in Infants and Children with Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency (Nokoff NJ, Buchanan C and Barker JM)
- Challenges in Adolescent and Adult Males with Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency (Claahsen-van der Grinten HL, Adriaansen BPH and Falhammar H)
- Clinical Manifestations and Challenges in Adolescent and Adult Females with Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency (Engberg H, Nordenstrӧm A and Hirschberg AL)
- Mental Health Issues Associated with Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency, (Sandberg DE, Gardner M and Lapham ZK)
- Life With Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency: Challenges and Burdens (Witchel SF, Miller T, McCann E and Gupta A)
- Barriers to the Management of Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency (Eitel KB and Fechner PY)
- Future Directions in the Management of Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency (Sarafoglou K and Auchus RJ)
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 is a trademark 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. 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, including the extent to which patients and physicians accept and adopt CRENESSITY; whether CRENESSITY receives adequate reimbursement from third-party payors; 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 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-0018 01/2025
View original content to download multimedia:https://www.prnewswire.com/news-releases/neurocrine-biosciences-announces-classic-congenital-adrenal-hyperplasia-supplement-published-today-in-the-journal-of-clinical-endocrinology--metabolism-302356465.html
SOURCE Neurocrine Biosciences, Inc.
FAQ
What is the significance of NBIX's CRENESSITY for CAH treatment?
What topics does the NBIX-sponsored CAH supplement in JCEM cover?
How does NBIX's CRENESSITY work in treating CAH?
What is the purpose of NBIX's publication in The Journal of Clinical Endocrinology & Metabolism?
