A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment

This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 28 weeks in approximately 81 pediatric participants with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The study consists of a 28-week double blind, placebo-controlled period, followed by 24 weeks of open-label treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 14 months for the core study and will be a variable amount of time per participant for the OLE (estimated to be approximately 3 years).

Start Date25 Jun 2021

Sponsor / Collaborator

Neurocrine Biosciences, Inc.

NCT04490915

/ Active, not recruitingPhase 3

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment

This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 24 weeks in approximately 165 adult participants with classic CAH due to 21-hydroxylase deficiency. The study consists of a 24-week randomized, double-blind, placebo-controlled period, followed by 1 year of active treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 20 months for the core study and will be a variable amount of time per participant for the OLE (estimated to be approximately 3 years).

Start Date16 Dec 2020

Sponsor / Collaborator

Neurocrine Biosciences, Inc.

NCT04045145

/ CompletedPhase 2

A Phase 2, Open-Label, Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Pediatric Subjects With Congenital Adrenal Hyperplasia

This is a Phase 2, open-label, multiple-dose, study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-74788 (crinecerfont) in pediatric participants (14 to 17 years of age) with a documented medical diagnosis of classic 21-hydroxylase deficiency congenital adrenal hyperplasia (CAH).

Start Date12 Dec 2019

Sponsor / Collaborator

Neurocrine Biosciences, Inc.

100 Clinical Results associated with Crinecerfont

100 Translational Medicine associated with Crinecerfont

100 Patents (Medical) associated with Crinecerfont

Literatures (Medical) associated with Crinecerfont

29 Jan 2025·JOURNAL OF PEDIATRIC ENDOCRINOLOGY & METABOLISM

Article

Author: Kamrath, Clemens ; Claahsen-van der Grinten, Hedi L.

Abstract:

21-Hydroxylase deficiency (21OHD), the most common form of congenital adrenal hyperplasia (CAH), leads to impaired cortisol synthesis and androgen excess. Current treatments of patients with classic 21OHD with supraphysiological doses of glucocorticoids pose risks such as impaired growth and metabolic complications. We discuss the CRH receptor antagonist as a therapeutic option for children with classic 21OHD. A phase three trial of crinecerfont, a CRH receptor antagonist, offers a promising new treatment option. Crinecerfont helped to reduce glucocorticoid doses and to lower androgen levels. However, the study population may not be fully representative of the general 21OHD population. Successful implementation depends on patient adherence and monitoring to avoid possible complications such as adrenal crises. Overall, crinecerfont represents a valuable development, but further research and careful clinical management are needed to optimize its use in CAH treatment.

21 Jan 2025·JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM

Review

Author: Auchus, Richard J ; Sarafoglou, Kyriakie

Abstract:

Context:

The traditional management of classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency (21OHD) is difficult and often suboptimal.

Objective:

To review improvements in the diagnosis and management of 21OHD.

Design:

Literature review, synthesis, and authors’ experience.

Setting:

United States (2 centers).

Participants:

Not applicable.

Interventions:

Not applicable.

Main Outcomes:

Not applicable.

Results:

The 11-oxygenated androgens are abundant in 21OHD, and their measurement might improve diagnosis and medication titration. Several new treatments are under development.

Conclusion:

Circadian delivery of hydrocortisone improves disease management of 21OHD compared to conventional glucocorticoids. Glucocorticoid-sparing therapies such as crinecerfont and atumelnant offer the potential for a block-and-replace strategy, with physiologic replacement dosing of hydrocortisone.

Clinical Trial Registration:

None.

01 Jan 2025·Revue medicale suisse

Endocrinologie : ce qui a changé en 2024

Article

Author: Cruchon, Karim ; Papadakis, Georgios ; Giordani, Ilaria ; Ameti, Adelina ; Sykiotis, Gerasimos P

In this article, we look at a selection of recent developments in various areas of endocrinology. We focus on advances in endocrine pharmacotherapy and endocrine surgery, addressing several areas: a) the thyroid safety of Glucagon-Like Peptide-1 (GLP1) analogues; b) the efficacy of adrenal surgery for mild autonomous cortisol secretion; c) crinecerfont in the management of congenital adrenal hyperplasia in adults and children; d) paltusotin as a novel oral therapy for acromegaly and e) TransCon PTH (palopegteriparatide) as a novel therapy for chronic hypoparathyroidism.

News (Medical) associated with Crinecerfont

21 Jan 2025

·www.prnewswire.com

Neurocrine Biosciences Announces Classic Congenital Adrenal Hyperplasia Supplement Published Today in The Journal of Clinical Endocrinology & Metabolism

SAN DIEGO, Jan. 21, 2025 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced publication of a classic congenital adrenal hyperplasia (CAH)-focused supplement in The Journal of Clinical Endocrinology & Metabolism (JCEM), sponsored by the company. The supplement, titled "Challenges and Opportunities in the Management of Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency Throughout the Lifetime," contains eight review articles that provide a comprehensive look at the clinical, psychosocial, treatment-related and day-to-day challenges faced by individuals with classic CAH. JCEM is a leader in disseminating research that supports healthcare providers, patients and caregivers in advancing the understanding and management of various endocrinology conditions, such as CAH. "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 95% of CAH cases are caused by variants of the CYP21A2 gene that leads to deficiency of the enzyme 21-hydroxylase. Severe deficiency of this enzyme leads to an inability of the adrenal glands to produce enough cortisol and, in approximately 75% of cases, aldosterone. Because individuals with CAH are still able to produce androgens, the unused precursors that would normally be used to make cortisol instead result in the production of excess amounts of androgens. If left untreated, CAH can result in salt wasting, dehydration and even death. 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 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 SOURCE Neurocrine Biosciences, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug Approval

08 Jan 2025

·neurocrine.gcs-web.com

Neurocrine Biosciences Announces Publication on Traditional Glucocorticoid Treatment in Classic Congenital Adrenal Hyperplasia in Expert Review of Endocrinology & Metabolism

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 SAN DIEGO, Jan. 8, 2025 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced publication of a narrative review discussing the challenges of traditional treatment of classic congenital adrenal hyperplasia (CAH) with glucocorticoids (GCs) alone and the potential benefits of introducing novel non-GC mechanisms for treating the condition that may enable lower, more physiologic GC dosing. The publication appears in Expert Review of Endocrinology & Metabolism. 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 95% of CAH cases are caused by variants of the CYP21A2 gene that leads to deficiency of the enzyme 21-hydroxylase (21-OH). Severe deficiency of this enzyme leads to an inability of the adrenal glands to produce enough cortisol and, in approximately 75% of cases, aldosterone. Because individuals with CAH are still able to produce androgens, the unused precursors that would normally be used to make cortisol instead result in the production of excess amounts of androgens. If left untreated, CAH can result in salt wasting, dehydration and even death. 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 View original content to download multimedia:https://www.prnewswire.com/news-releases/neurocrine-biosciences-announces-publication-on-traditional-glucocorticoid-treatment-in-classic-congenital-adrenal-hyperplasia-in-expert-review-of-endocrinology--metabolism-302345127.html SOURCE Neurocrine Biosciences, Inc. Neurocrine Biosciences, Inc., Media: Aimee White, 1-858-354-7865, media@neurocrine.com. Investors: Todd Tushla, 1-858-617-7143, ir@neurocrine.com

23 Dec 2024

·www.prnewswire.com

Neurocrine Biosciences, Inc. Selects PANTHERx®Rare Specialty Pharmacy for Distribution of Crenessity™ (crinecerfont)

PITTSBURGH, Dec. 23, 2024 /PRNewswire/ -- PANTHERx® Rare, a leader in rare disease product patient access and support services in the United States, is pleased to announce that it was selected by Neurocrine Biosciences, Inc. for the distribution of CRENESSITY™ (crinecerfont), an oral selective corticotropin-releasing factor type 1 (CRF1) receptor antagonist indicated to improve androgen control and enable a reduced glucocorticoid dose in patients 4 years of age and older with classic congenital adrenal hyperplasia (CAH). CRENESSITY is the first non-steroidal treatment approved for classic CAH and a first-in-class therapy that provides a novel approach to treatment for this rare disorder. CAH is a rare, autosomal recessive genetic disorder that results from deficiency of one of the enzymes needed for adrenal hormone production. The most common cause of CAH is 21-hydroxylase (21-OHD) deficiency, which accounts for approximately 95% of cases.2 21-OHD deficiency prevents the body from producing cortisol, which is essential to respond to stress and illness. In about 75% of cases, aldosterone production is also affected, which impairs the body's ability to regulate salt and water levels, and thereby blood pressure.2,3 In response to low levels of cortisol and aldosterone, the hypothalamus-pituitary-adrenal (HPA) axis is activated, leading to overproduction of androgens.2 Excess androgen production can cause a variety of symptoms, including anxiety, acne, hirsutism, atypical genital development in affected females, fertility issues, early-onset puberty, and rapid growth followed by premature completion resulting in short stature.3 These symptoms can significantly impact both the physical and mental health of those affected with CAH. "We are proud to partner with Neurocrine Biosciences for the distribution of CRENESSITY. This collaboration underscores PANTHERx's dedication to providing personalized care and seamless access to transformative therapies for patients with rare and complex conditions," said Rob Snyder, Executive Chair, PANTHERx® Rare Pharmacy. About PANTHERx Rare PANTHERx Rare is a dual-accredited specialty pharmacy focused on rare and orphan disease, distributing several orphan products and providing access and support services to those needing them most. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to people living with rare and devastating conditions. While each rare condition affects few people, together all rare diseases impact an estimated 25 to 30 million Americans.3 Currently over 7,000 rare diseases have been identified and more than 90% of rare diseases are still without an FDA-approved treatment. 3 Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community. PANTHERx is a dual-accredited specialty pharmacy, holding distinctions in rare disease including the Accreditation Commission for Health Care (ACHC) Distinction in Orphan Drugs and Utilization Review Accreditation Commission (URAC) Rare Disease Center of Excellence. As a pharmacy focused on patient satisfaction, PANTHERx is now a six-time winner of the prestigious MMIT Patient Choice Award, including the 2023 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania and is licensed in all 50 states and US territories. 1. CRENESSITY™ (crinecerfont) [package insert]. Neurocrine Biosciences, Inc. 2024. 2. Congenital Adrenal Hyperplasia (CAH). Neurocrine Biosciences, Inc. Accessed October 21, 2024. 3. Congenital Adrenal Hyperplasia. NORD (National Organization for Rare Disorders). Published June 8, 2023. Accessed October 21, 2024. For more information, please contact [email protected] SOURCE PANTHERx Rare Pharmacy WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug ApprovalOrphan Drug

100 Deals associated with Crinecerfont

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Classic Congenital Adrenal Hyperplasia	US	Neurocrine Biosciences, Inc.	13 Dec 2024

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Congenital Adrenal Hyperplasia Due to 21 Hydroxylase Deficiency	Phase 3	US	Neurocrine Biosciences, Inc.	25 Jun 2021
Congenital Adrenal Hyperplasia Due to 21 Hydroxylase Deficiency	Phase 3	BE	Neurocrine Biosciences, Inc.	25 Jun 2021
Congenital Adrenal Hyperplasia Due to 21 Hydroxylase Deficiency	Phase 3	CA	Neurocrine Biosciences, Inc.	25 Jun 2021
Congenital Adrenal Hyperplasia Due to 21 Hydroxylase Deficiency	Phase 3	FR	Neurocrine Biosciences, Inc.	25 Jun 2021
Congenital Adrenal Hyperplasia Due to 21 Hydroxylase Deficiency	Phase 3	DE	Neurocrine Biosciences, Inc.	25 Jun 2021
Congenital Adrenal Hyperplasia Due to 21 Hydroxylase Deficiency	Phase 3	GR	Neurocrine Biosciences, Inc.	25 Jun 2021
Congenital Adrenal Hyperplasia Due to 21 Hydroxylase Deficiency	Phase 3	IT	Neurocrine Biosciences, Inc.	25 Jun 2021
Congenital Adrenal Hyperplasia Due to 21 Hydroxylase Deficiency	Phase 3	PL	Neurocrine Biosciences, Inc.	25 Jun 2021
Congenital Adrenal Hyperplasia Due to 21 Hydroxylase Deficiency	Phase 3	ES	Neurocrine Biosciences, Inc.	25 Jun 2021
Congenital Adrenal Hyperplasia Due to 21 Hydroxylase Deficiency	Phase 3	GB	Neurocrine Biosciences, Inc.	25 Jun 2021

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04806451 (CAHtalyst \| CAHtalyst Pediatric, CTgov)	Phase 3	Adrenal Hyperplasia, Congenital \| Congenital Adrenal Hyperplasia Due to 21 Hydroxylase Deficiency	103	Placebo+Crinecerfont (Placebo)	nvthsvifxc(bbxraouftt) = hzquogspgq nqygzznaxe (spprgqxebx, vujvutacqp - gaclhtmuqh)	-	05 Feb 2025
				Crinecerfont (Crinecerfont)	nvthsvifxc(bbxraouftt) = ssmstptjxf nqygzznaxe (spprgqxebx, huabigwyxw - vetamtqhyo)
NCT04806451 (FDA_CDER) Manual	Phase 3	Classic Congenital Adrenal Hyperplasia	103	CRENESSITY (Pediatric)	cuxraheitp(rsiqiylcbp) = bzjmczzujy xsntctgwzx (yowqeeeyyi ) View more	Positive	13 Dec 2024
				Placebo (Pediatric)	cuxraheitp(rsiqiylcbp) = vrmrorjorv xsntctgwzx (yowqeeeyyi ) View more
NCT04490915 (FDA_CDER) Manual	Phase 3	Classic Congenital Adrenal Hyperplasia	182	CRENESSITY (Adults)	lptiylyzpi(yfqnrbmeng) = hbzoatzife royltxrihq (jielmaavzi ) View more	Positive	13 Dec 2024
				Placebo (Adults)	lptiylyzpi(yfqnrbmeng) = atqzzclxqv royltxrihq (jielmaavzi ) View more
NCT04045145 (CTgov)	Phase 2	Adrenal Hyperplasia, Congenital	8	Crinecerfont	lmsrynauay(oxvsbjblzf) = jhobazlqkf ckuqgdgbct (yfopihnoyu, nuwcajooij - kkrpwnbdft) View more	-	18 Jul 2024
NCT04806451 (CAHtalyst, Pubmed)	Phase 3	Adrenal Hyperplasia, Congenital androstenedione	103	Crinecerfont	puktqqkant(ibevwwaatg) = jtmxbaspdr esuydkcndi (qabjjsjdmx, -6.9)	Positive	02 Jun 2024
				Placebo	puktqqkant(ibevwwaatg) = bvxftiifbx esuydkcndi (qabjjsjdmx, 2.5) View more
NCT04490915 (CAHtalyst, Pubmed)	Phase 3	Adrenal Hyperplasia, Congenital 21-hydroxylase deficiency	182	Crinecerfont	mxizgsmwpq(qlyvsycxdl) = Fatigue were the most common adverse events in the two trial groups ixptbzcoyy (pbyqzoajbr ) View more	Positive	01 Jun 2024
				Placebo
NCT04806451 (CAHtalyst Pediatric, ENDO2024)	Phase 3	Adrenal Hyperplasia, Congenital 21-hydroxylase deficiency \| ACTH \| androstenedione ... View more	103	Crinecerfont	rzzzfqwall(ojmedkdpni) = dpyxaegoau dfkypusfsk (rtsvsfhrki ) View more	Positive	01 Jun 2024
NCT04490915 (CAHtalyst, ENDO2024)	Phase 3	Adrenal Hyperplasia, Congenital androstenedione	182	Crinecerfont 100 mg BID	biilzojmlb(cceiiqvozi) = oyrxebtxie hcybzgwrqt (wbvickgycz ) View more	Positive	01 Jun 2024
				Placebo	sglujkofoh(ticujrmjsl) = sgqccvvrga uvjfzusnby (nknyptewgv )
NCT04806451 \| NCT04490915 (CAHtalyst, PRNewswire) Manual	Phase 3	Adrenal Hyperplasia, Congenital	-	crinecerfont	ormirquinh(bebkxgpqoe) = a statistically significant decrease in serum androstenedione from baseline with crinecerfont at Week 4 over baseline, while both studies showed crinecerfont treatment led to statistically significant reductions in daily glucocorticoid from baseline while maintaining androgen control at Week 28 and Week 24, respectively byemzhgmzc (bwrmyhbvcb ) View more	Positive	05 Dec 2023
NCT03525886 (CTgov)	Phase 2	Adrenal Hyperplasia, Congenital	18	NBI-74788 (Cohort 1 (50 mg QHS))	kszldawvkc(ppsrjgaxsm) = pksgrhixpv hfjiparwzt (nmhythehcm, adgqtmvejz - pgoqeluatz) View more	-	03 May 2022
				NBI-74788 (Cohort 2 (100 mg QHS))	kszldawvkc(ppsrjgaxsm) = vygqcyqaxe hfjiparwzt (nmhythehcm, vpwhocwlvz - etmhomlsld) View more

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