Neurocrine Biosciences Announces FDA Approval of CRENESSITY™ (crinecerfont), a First-in-Class Treatment for Children and Adults With Classic Congenital Adrenal Hyperplasia
Neurocrine Biosciences (NBIX) has received FDA approval for CRENESSITY™ (crinecerfont), the first new treatment in 70 years for classic congenital adrenal hyperplasia (CAH). The drug is approved as an adjunctive treatment to glucocorticoid replacement for patients aged 4 and older.
The approval is supported by the largest-ever CAH clinical trial program, with significant results in both pediatric and adult studies. In pediatric trials, CRENESSITY showed 4x greater steroid dose reduction versus placebo. In adult trials, 63% of patients achieved glucocorticoid dose reduction while maintaining or improving androgen levels, compared to 18% on placebo.
The drug will be commercially available within a week through PANTHERx Rare pharmacy, with most patients expected to pay $10 or less monthly.
Neurocrine Biosciences (NBIX) ha ricevuto l'approvazione della FDA per CRENESSITY™ (crinecerfont), il primo nuovo trattamento in 70 anni per l'iperplasia surrenalica congenita classica (CAH). Il farmaco è approvato come trattamento adiuvante per la sostituzione dei glucocorticoidi per pazienti di età pari o superiore a 4 anni.
L'approvazione è supportata dal più grande programma di studi clinici mai realizzato sulla CAH, con risultati significativi sia negli studi pediatrici che in quelli per adulti. Negli studi pediatrici, CRENESSITY ha mostrato una riduzione della dose di steroidi 4 volte superiore rispetto al placebo. Negli studi per adulti, il 63% dei pazienti ha raggiunto una riduzione della dose di glucocorticoidi mantenendo o migliorando i livelli di androgeni, rispetto al 18% del gruppo placebo.
Il farmaco sarà disponibile commercialmente entro una settimana attraverso la farmacia PANTHERx Rare, con la maggior parte dei pazienti che ci si aspetta pagheranno $10 o meno al mese.
Neurocrine Biosciences (NBIX) ha recibido la aprobación de la FDA para CRENESSITY™ (crinecerfont), el primer nuevo tratamiento en 70 años para la hiperplasia adrenal congénita clásica (CAH). El medicamento está aprobado como tratamiento complementario a la sustitución de glucocorticoides para pacientes de 4 años en adelante.
La aprobación está respaldada por el programa de ensayos clínicos más grande jamás realizado para la CAH, con resultados significativos tanto en estudios pediátricos como para adultos. En los ensayos pediátricos, CRENESSITY mostró una reducción de la dosis de esteroides 4 veces mayor en comparación con el placebo. En ensayos para adultos, el 63% de los pacientes logró una reducción de la dosis de glucocorticoides manteniendo o mejorando los niveles de andrógenos, en comparación con el 18% en el grupo placebo.
El medicamento estará disponible comercialmente en una semana a través de la farmacia PANTHERx Rare, y se espera que la mayoría de los pacientes paguen $10 o menos al mes.
Neurocrine Biosciences (NBIX)는 CRENESSITY™ (crinecerfont)에 대해 FDA 승인을 받았습니다. 이는 고전적인 선천적 부신 과형성증 (CAH)을 위한 70년 만의 첫 번째 새로운 치료법 입니다. 이 약물은 4세 이상의 환자를 위한 글루코코르티코이드 대체 치료의 보조 치료제로 승인되었습니다.
이번 승인은 CAH 임상 시험 프로그램 중 가장 큰 규모로 이루어졌으며, 소아와 성인 연구 모두에서 중요한 결과가 도출되었습니다. 소아 시험에서는 CRENESSITY가 위약에 비해 스테로이드 용량을 4배 줄이는 효과를 보였습니다. 성인 시험에서는 63%의 환자가 안드로겐 수치를 유지하거나 개선하며 글루코코르티코이드 용량을 줄일 수 있었고, 이와 대조적으로 위약 그룹에서는 18%만이 그러한 효과를 보였습니다.
이 약물은 PANTHERx Rare 약국을 통해 일주일 이내에 상용화될 예정이며, 대부분의 환자들은 월 $10 이하의 비용을 지불할 것으로 예상됩니다.
Neurocrine Biosciences (NBIX) a reçu l'approbation de la FDA pour CRENESSITY™ (crinecerfont), le premier nouveau traitement en 70 ans pour l'hyperplasie surrénale congénitale classique (CAH). Le médicament est approuvé comme traitement adjuvant à la substitution glucocorticoïde pour les patients âgés de 4 ans et plus.
Cette approbation est soutenue par le plus grand programme d'essais cliniques jamais réalisé sur la CAH, avec des résultats significatifs tant dans les études pédiatriques que pour les adultes. Dans les études pédiatriques, CRENESSITY a montré une réduction de la dose de stéroïdes quatre fois supérieure à celle du placebo. Dans les études pour adultes, 63 % des patients ont réussi à réduire leur dose de glucocorticoïdes tout en maintenant ou améliorant leurs niveaux d'androgènes, contre 18 % dans le groupe placebo.
Le médicament sera disponible à la vente dans une semaine via la pharmacie PANTHERx Rare, avec la plupart des patients s'attendant à payer 10 $ ou moins par mois.
Neurocrine Biosciences (NBIX) hat die FDA-Zulassung für CRENESSITY™ (crinecerfont) erhalten, das erste neue Medikament seit 70 Jahren zur Behandlung der klassischen angeborenen Adrenalhyperplasie (CAH). Das Medikament ist als Zusatztherapie zur Glukokortikoid-Substitution für Patienten im Alter von 4 Jahren und älter zugelassen.
Die Zulassung stützt sich auf das bislang größte klinische Prüfprogramm zur CAH, mit signifikanten Ergebnissen sowohl in der Kinder- als auch in der Erwachsenenstudie. In den pädiatrischen Studien zeigte CRENESSITY eine vierfach höhere Reduktion der Steroiddosis im Vergleich zu Placebo. In den Studien an Erwachsenen erreichten 63% der Patienten eine Reduzierung der Glukokortikoiddosis, während sie die Androgenspiegel aufrechterhielten oder verbesserten, im Vergleich zu 18% in der Placebogruppe.
Das Medikament wird innerhalb einer Woche über die PANTHERx Rare Apotheke für den Handel verfügbar sein, und die meisten Patienten werden voraussichtlich monatlich 10 $ oder weniger bezahlen.
- First new CAH treatment approved in 70 years, establishing market leadership
- Strong clinical trial results showing 4x greater steroid dose reduction in pediatric patients
- 63% efficacy rate in adult patients vs 18% for placebo
- Rare Pediatric Disease Priority Review Voucher granted
- Accessible pricing strategy with most patients paying $10 or less monthly
- Two patients (1.6%) in adult trials experienced adrenal crisis
- Multiple adverse reactions reported including fatigue, headache, and dizziness
- Requires ongoing glucocorticoid treatment as adjunctive therapy
Insights
The FDA approval of CRENESSITY represents a groundbreaking advancement in CAH treatment after a 70-year gap. This first-in-class CRF1 receptor antagonist demonstrates impressive clinical efficacy in both pediatric and adult populations. The drug's ability to reduce excess ACTH and androgen production while enabling glucocorticoid dose reduction addresses a critical unmet need in CAH management.
The robust clinical data from the CAHtalyst trials shows significant improvements:
This approval marks a significant commercial opportunity for Neurocrine Biosciences in the rare disease space. As the first new CAH treatment in 70 years, CRENESSITY enjoys first-mover advantage and potential pricing power in this underserved market. The receipt of a Rare Pediatric Disease Priority Review Voucher adds substantial value, as these vouchers typically sell for
The comprehensive patient support program and specialty pharmacy distribution through PANTHERx Rare should facilitate market penetration. With strong efficacy data published in The New England Journal of Medicine and a patient-friendly cost structure, CRENESSITY could become a significant revenue driver for NBIX, potentially achieving blockbuster status in this rare disease market.
- CRENESSITY, the first new treatment available in 70 years to the classic congenital adrenal hyperplasia (CAH) community, offers a paradigm-shifting treatment approach
- FDA approval supported by data from the largest-ever clinical trial program in pediatric and adult patients with classic CAH
- CRENESSITY is expected to be commercially available in approximately one week
- Rare Pediatric Disease Priority Review Voucher granted in connection with approval
"For the last three decades, Neurocrine Biosciences, together with our late founder, Wylie W. Vale, has conducted groundbreaking research uncovering the critical role of corticotropin-releasing factor and its receptor, CRF1, in the pathophysiology of congenital adrenal hyperplasia," said Kyle W. Gano, Ph.D., Chief Executive Officer, Neurocrine Biosciences. "The approval of CRENESSITY is a significant milestone for the CAH community, and we are grateful to the individuals who participated in our clinical trials, including their families and caregivers, and to the clinical investigators who helped advance a new therapy and class of medicines."
"Patients and families struggle to achieve balance between managing the symptoms of CAH and the side effects or complications of treatment with high-dose steroids, which may impact quality of life," said Dina Matos, Executive Director, CARES Foundation. "We are grateful to Neurocrine Biosciences for engaging with our community throughout the drug development process to understand our needs and ultimately providing this new medication that can help reduce excess adrenal androgens and the need for high-dose steroid treatment for individuals living with CAH."
CRENESSITY is expected to be commercially available in approximately one week. The medication will be provided through PANTHERx Rare, a specialty pharmacy, to centralize and simplify CRENESSITY prescription fulfillment.
Neurocrine Biosciences is committed to supporting patients in obtaining treatment with CRENESSITY by offering Neurocrine Access Support, a free, comprehensive assistance program created for patients, caregivers and healthcare providers. It offers a range of options to make sure patients have everything they need to begin and continue taking CRENESSITY. A dedicated Care Coordinator, backed by a team, is available to help patients and caregivers navigate the insurance process and identify appropriate financial assistance options. Most patients will pay
*Additional terms and conditions apply.
CAHtalyst™ Clinical Program Overview:
The FDA approval is supported by the largest-ever clinical trial program of classic CAH, the CAHtalyst Pediatric and Adult Phase 3 global registrational studies. CAHtalyst Phase 3 data results in pediatric and adult patients with classic CAH were published in The New England Journal of Medicine.
"The clinical results across both CAHtalyst studies support the efficacy and safety profile of CRENESSITY and its ability to reduce the overproduction of adrenal androgens, allowing for a meaningful reduction in glucocorticoid dosage, while maintaining or enhancing control of these androgens," said Richard Auchus, M.D., Ph.D., Professor, University of Michigan Health, Principal Investigator. "Chronic treatment with supraphysiologic glucocorticoids can cause a number of short- and long-term health consequences, such as obesity, hypertension and osteoporosis, so the ability for patients with CAH to lower their glucocorticoid dose to a more physiologic level can have profound benefits."
In both CAHtalyst studies, CRENESSITY enabled lower steroid doses and decreased androgen levels.
Phase 3 CAHtalyst Pediatric Study:
- The CAHtalyst Pediatric study met its primary endpoint, with CRENESSITY significantly decreasing androstenedione levels from baseline to Week 4 versus patients taking placebo who experienced a substantial increase in androstenedione levels.
- Children taking CRENESSITY were also able to significantly reduce their GC doses at Week 28 while maintaining or improving androgen levels, a key secondary endpoint.
- Children taking CRENESSITY saw approximately four times greater reduction in androstenedione compared with those taking placebo.
- Approximately four times greater steroid dose reduction in children taking CRENESSITY was seen compared with those taking placebo.
- Children taking CRENESSITY saw approximately 12 times greater reduction in 17-hydroxyprogesterone (17-OHP) compared with those taking placebo.
- Headache, abdominal pain, fatigue, nasal congestion and nosebleed were the most common adverse drug reactions (ADRs) among the pediatric population treated with CRENESSITY. Most side effects were temporary and mild to moderate in severity.
Phase 3 CAHtalyst Adult Study:
- The CAHtalyst Adult study met its primary endpoint with CRENESSITY enabling significant GC dose reductions at Week 24 (while maintaining or improving baseline androstenedione levels) and key secondary endpoint of decreasing androstenedione levels at Week 4.
- A significantly higher number of patients taking CRENESSITY (
63% ) achieved a GC dose in the physiologic range while androstenedione was maintained or improved compared with patients taking placebo (18% ). - Approximately two times greater steroid dose reduction was seen in people taking CRENESSITY compared with those taking placebo.
- People taking CRENESSITY saw an eight times greater reduction in androstenedione compared with those taking placebo.
- People taking CRENESSITY saw a 37 times greater reduction in 17-OHP compared with those taking placebo.
- Fatigue, headache, dizziness, joint pain, back pain, decreased appetite and muscle pain were the most common ADRs in the CRENESSITY treatment group. Most side effects were temporary and mild to moderate in severity.
CRENESSITY was well tolerated with few treatment-related adverse events in both CAHtalyst studies. Pediatric and adult patients taking CRENESSITY had no treatment-related serious adverse events.
Adrenal insufficiency and crisis are risks of living with CAH that CRENESSITY does not address and can occur when a patient's GC dose is too low. In the CAHtalyst Pediatric study, there were no cases of adrenal crisis among patients taking CRENESSITY or placebo. In the CAHtalyst Adult study, two patients (
For more information about CRENESSITY, visit Crenessity.com.
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 The CAHtalyst™ Studies
The Phase 3 CAHtalyst™ global registrational studies were designed to evaluate the safety, efficacy and tolerability of CRENESSITY in children and adults with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The CAHtalyst studies were the largest-ever clinical trial program in classic CAH, including 285 pediatric and adult patients.
The CAHtalyst Pediatric study included 103 pediatric patients aged four to 17 years. The study tested two questions. The first question evaluated whether four weeks of CRENESSITY treatment could improve androgen control. The second question evaluated whether an additional 24 weeks of CRENESSITY treatment enabled customized glucocorticoid (GC) down-titration while androstenedione levels were maintained or improved. The CAHtalyst Adult study included 182 adult patients aged 18 to 58 years. Similarly, the first question of the study evaluated whether four weeks of CRENESSITY treatment could improve androgen control, and the second question evaluated whether an additional 20 weeks of CRENESSITY treatment enabled GC reduction to physiologic range while androstenedione levels were maintained or improved.
Data from the CAHtalyst Phase 3 studies supported approval of CRENESSITY by the
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, but few options. 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) due to 21-hydroxylase deficiency; 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.
© 2024 Neurocrine Biosciences, Inc. All Rights Reserved. CP-CFT-US-0070 12/2024
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FAQ
When will CRENESSITY (NBIX) be commercially available?
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How significant was the steroid dose reduction in CRENESSITY pediatric trials?
