This section highlights FDA-related milestones and regulatory updates for drugs developed by Neurocrine Biosciences (NBIX).
Over the past two years, Neurocrine Biosciences has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
CAHtalyst™, crinecerfont, INGREZZA, NBI-1065845, NBI-1117567, NBI-1117568, and NBI-1140675. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
CAHtalyst™ - FDA Regulatory Timeline and Events
CAHtalyst™ is a drug developed by Neurocrine Biosciences for the following indication: For the Treatment of Congenital Adrenal Hyperplasia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- CAHtalyst™
- Announced Date:
- May 8, 2025
- Indication:
- For the Treatment of Congenital Adrenal Hyperplasia
Announcement
Neurocrine Biosciences, Inc. nnounced new results from subgroup analyses of the Phase 3 CAHtalyst™ Pediatric study.
AI Summary
Neurocrine Biosciences recently shared new subgroup analysis results from the Phase 3 CAHtalyst Pediatric study. The study focused on children with classic congenital adrenal hyperplasia and found that patients taking CRENESSITY (crinecerfont) were able to maintain or improve their androstenedione levels while reducing glucocorticoid doses. This benefit was consistent across all subgroups, including variations in demographic factors, baseline hormone levels, and initial glucocorticoid doses. Reducing glucocorticoid treatment is important because high doses can lead to significant side effects and long-term health issues. The findings suggest that CRENESSITY offers a safer way to manage adrenal androgen levels and improve care for young patients with CAH. These promising results will be presented at the 2025 Joint Congress of the European Society for Paediatric Endocrinology and the European Society of Endocrinology.
Read Announcement- Drug:
- CAHtalyst™
- Announced Date:
- June 3, 2024
- Indication:
- For the Treatment of Congenital Adrenal Hyperplasia
Announcement
Neurocrine Biosciences, Inc. announced that the primary study results from its CAHtalyst™ Phase 3 study investigating crinecerfont for the treatment of adults ages 18 and older with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency have been published in The New England Journal of Medicine online edition and will appear in a future print issue of the journal.
AI Summary
Neurocrine Biosciences, Inc. announced that the primary results from its CAHtalyst™ Phase 3 study of crinecerfont in adults with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency have been published online in The New England Journal of Medicine and will appear in an upcoming print issue. The study met its primary and key secondary endpoints by showing that crinecerfont can help reduce the need for high glucocorticoid doses while maintaining proper androgen control. This breakthrough offers hope for a new treatment approach that could decrease the dose-related side effects of steroids in CAH patients. The results mark an important milestone in advancing treatment options for adults with CAH and could lead to improved management of this challenging condition over the long term.
Read Announcement- Drug:
- CAHtalyst™
- Announced Date:
- May 28, 2024
- Indication:
- For the Treatment of Congenital Adrenal Hyperplasia
Announcement
Neurocrine Biosciences, Inc. announced that it will present key information from its neuroendocrinology pipeline, including new Phase 3 clinical study data from its CAHtalyst™ registrational studies of crinecerfont in pediatric and adult patients with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency and data from its modified-release hydrocortisone (MRHC) studies in primary adrenal insufficiency and CAH.
AI Summary
Neurocrine Biosciences recently announced that it will present key data from its neuroendocrinology pipeline at the upcoming Endocrine Society Annual Meeting in Boston. The company will share new Phase 3 clinical study results from its CAHtalyst™ registrational trials, which tested crinecerfont in both pediatric and adult patients with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. These studies evaluated the safety, efficacy, and tolerability of crinecerfont, aiming to offer a treatment that may help lower the need for high glucocorticoid doses and reduce associated side effects. In addition, the presentation will include data from studies investigating modified-release hydrocortisone (MRHC) in patients with primary adrenal insufficiency and CAH. The findings could provide important insights into improving therapies and reducing long-term complications for patients with these challenging endocrine disorders.
Read Announcement- Drug:
- CAHtalyst™
- Announced Date:
- May 14, 2024
- Indication:
- For the Treatment of Congenital Adrenal Hyperplasia
Announcement
Neurocrine Biosciences, Inc. and Diurnal Ltd., a Neurocrine Biosciences company, presented baseline data from the CAHtalyst™ Phase 3 studies of crinecerfont in adult and pediatric patients with congenital adrenal hyperplasia (CAH), and modified-release hydrocortisone (Chronocort®) data for a Phase 2 clinical study (CHAMPAIN) in participants with primary adrenal insufficiency and in a Phase 3 extension study in CAH. These data, along with several additional posters were presented at the European Congress of Endocrinology 2024 meeting in Stockholm.
AI Summary
Neurocrine Biosciences, Inc. and Diurnal Ltd., a Neurocrine Biosciences company, presented important new data at the European Congress of Endocrinology 2024 meeting in Stockholm. The companies shared baseline data from the CAHtalyst™ Phase 3 studies, which evaluated crinecerfont in both adult and pediatric patients with congenital adrenal hyperplasia (CAH). These studies are significant as they help determine the effectiveness of crinecerfont in reducing the symptoms and complications associated with CAH. Additionally, modified-release hydrocortisone (Chronocort®) data was presented from a Phase 2 clinical study called CHAMPAIN, which evaluated the treatment in participants with primary adrenal insufficiency. There was also data from a Phase 3 extension study in CAH patients. This new information could pave the way for improved treatment options for those suffering from CAH and primary adrenal insufficiency.
Read Announcement- Drug:
- CAHtalyst™
- Announced Date:
- May 3, 2024
- Indication:
- For the Treatment of Congenital Adrenal Hyperplasia
Announcement
Neurocrine Biosciences, Inc. today presented the CAHtalyst™ Pediatric Phase 3 clinical study baseline characteristics data for children and adolescents with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency enrolled in the study, along with CAHtalog™ Registry data assessing glucocorticoid treatment patterns in pediatric and adult patients with CAH.
AI Summary
Neurocrine Biosciences presented new data at the Pediatric Endocrine Society 2024 Annual Meeting, highlighting the challenges in treating congenital adrenal hyperplasia (CAH). The CAHtalyst™ Pediatric Phase 3 study enrolled 103 children and adolescents aged 4 to 17 with CAH due to 21-hydroxylase deficiency. Many participants showed signs of adrenal androgen excess, even with high, supraphysiologic doses of glucocorticoids. Common issues included obesity, advanced bone age, and early puberty, pointing to the need for better treatment options for young patients. In addition, the CAHtalog™ Registry data reviewed glucocorticoid treatment patterns in both pediatric and adult patients with CAH, revealing that many receive doses at or above the upper recommended limits. These findings emphasize the ongoing challenges in managing CAH and the importance of developing novel therapeutic strategies.
Read Announcement
crinecerfont - FDA Regulatory Timeline and Events
crinecerfont is a drug developed by Neurocrine Biosciences for the following indication: For the Treatment of Congenital Adrenal Hyperplasia (CAH).
This drug is approved by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- crinecerfont
- Announced Date:
- May 16, 2025
- Indication:
- For the Treatment of Congenital Adrenal Hyperplasia (CAH)
Announcement
Neurocrine Biosciences, Inc. announced it will present new data from the Phase 3 CAHtalyst™ Pediatric study showing lasting reductions in glucocorticoid doses in pediatric patients with classic congenital adrenal hyperplasia who received CRENESSITY™ (crinecerfont) for up to one year.
AI Summary
Neurocrine Biosciences, Inc. announced new data from its Phase 3 CAHtalyst™ Pediatric study, which focused on children with classic congenital adrenal hyperplasia. The study showed that treatment with CRENESSITY™ (crinecerfont) for up to one year led to lasting reductions in glucocorticoid doses. Even with lower glucocorticoid doses, key hormone levels such as ACTH, 17-hydroxyprogesterone, and androstenedione stayed below the initial baseline levels. These results suggest that CRENESSITY may help balance hormone levels while reducing the need for high-dose steroids.
In addition to improved hormone control, the study noted positive changes in clinical outcomes, including improvements in body mass index and insulin resistance. These findings highlight CRENESSITY’s potential to offer a more physiologic treatment regimen for pediatric patients with classic congenital adrenal hyperplasia, potentially reducing the risk of adverse side effects linked to high glucocorticoid doses.
Read Announcement- Drug:
- crinecerfont
- Announced Date:
- May 15, 2025
- Indication:
- For the Treatment of Congenital Adrenal Hyperplasia (CAH)
Announcement
Neurocrine Biosciences, announced new data from the Phase 3 CAHtalyst™ Adult and Pediatric studies of CRENESSITY™ (crinecerfont).
AI Summary
Neurocrine Biosciences recently announced new results from the Phase 3 CAHtalyst Adult and Pediatric studies for CRENESSITY™ (crinecerfont). In pediatric patients with classic congenital adrenal hyperplasia (CAH), 90% of those treated with CRENESSITY achieved significant improvements by reaching a key threshold for androstenedione reduction or lowering glucocorticoid doses, compared to only 21% of patients on placebo. These findings highlight that many children attained more physiologic glucocorticoid dosing while maintaining better hormone balance.
In adult males, the studies also revealed promising outcomes. Patients experienced improvements in select reproductive hormone levels, including normalization of luteinizing hormone and the androstenedione/testosterone ratio, despite substantial glucocorticoid dose reductions. This dual benefit suggests that CRENESSITY may help control excess androgen production while enabling lower glucocorticoid doses, potentially reducing the side effects associated with high-dose steroid therapy.
Read Announcement- Drug:
- crinecerfont
- Announced Date:
- December 20, 2024
- Indication:
- For the Treatment of Congenital Adrenal Hyperplasia (CAH)
Announcement
Neurocrine Biosciences, announced CRENESSITY™ (crinecerfont) is now commercially available in the United States. CRENESSITY was recently approved by the U.S. Food and Drug Administration as an adjunctive treatment to glucocorticoid replacement to control androgens in adult and pediatric patients four years of age and older with classic congenital adrenal hyperplasia (CAH).
AI Summary
Neurocrine Biosciences announced that CRENESSITY™ (crinecerfont) is now available for patients in the United States. The U.S. Food and Drug Administration approved the drug as an adjunctive treatment to glucocorticoid replacement. This approval is for managing androgen levels in both adult and pediatric patients, ages four and older, who suffer from classic congenital adrenal hyperplasia (CAH).
CRENESSITY is a first-in-class, oral treatment that reduces the production of adrenocorticotropic hormone (ACTH) and adrenal androgens. By doing so, it helps patients lower their glucocorticoid doses while effectively maintaining or improving hormone control. The medication is exclusively available through PANTHERx Rare, where CAH-trained pharmacists are accessible 24/7 to support patients, caregivers, and healthcare providers with all prescription needs.
Read Announcement- Drug:
- crinecerfont
- Announced Date:
- December 13, 2024
- Indication:
- For the Treatment of Congenital Adrenal Hyperplasia (CAH)
Announcement
Neurocrine Biosciences, announced the U.S. Food and Drug Administration has approved CRENESSITY™ (crinecerfont) capsules and oral solution as an adjunctive treatment to glucocorticoid replacement to control androgens in adult and pediatric patients four years of age and older with classic congenital adrenal hyperplasia (CAH), a rare, serious and lifelong genetic condition involving the adrenal glands.
AI Summary
Neurocrine Biosciences has announced that the U.S. FDA approved CRENESSITY™ (crinecerfont) capsules and oral solution as an adjunctive treatment to glucocorticoid replacement therapy. This approval is for controlling elevated androgen levels in both adult and pediatric patients (four years and older) suffering from classic congenital adrenal hyperplasia (CAH), a rare and lifelong genetic disorder affecting the adrenal glands. CRENESSITY works by selectively blocking the corticotropin-releasing factor type 1 receptor, which leads to a reduction in excessive adrenocorticotropic hormone (ACTH) and subsequent adrenal androgen production. By lowering these hormone levels, patients can potentially reduce the high glucocorticoid doses typically required, thereby lessening adverse side effects. This breakthrough treatment offers a new approach to CAH therapy, providing much-needed relief for patients and their families, and is expected to be commercially available soon.
Read Announcement- Drug:
- crinecerfont
- Announced Date:
- July 1, 2024
- Estimated Event Date Range:
- December 30, 2024 - December 30, 2024
- Target Action Date:
- December 30, 2024
- Indication:
- For the Treatment of Congenital Adrenal Hyperplasia (CAH)
Announcement
Neurocrine Biosciences, Inc announced that The agency set Prescription Drug User Fee (PDUFA) target action dates of December 29 and December 30, 2024, respectively..
AI Summary
Neurocrine Biosciences, Inc. announced that the U.S. Food and Drug Administration has accepted its two New Drug Applications (NDAs) for crinecerfont, aimed at treating congenital adrenal hyperplasia (CAH) in children, adolescents, and adults. The FDA has set Prescription Drug User Fee Act (PDUFA) target action dates of December 29 and December 30, 2024, for the capsule and oral solution formulations, respectively. This Priority Review designation speeds up the FDA’s evaluation process by four months, reflecting the urgent need for a treatment that offers significant benefits over current therapies for CAH.
If approved, crinecerfont would become the first new treatment for CAH in 70 years, potentially reducing the need for high-dose glucocorticoids and offering improved safety for patients. Neurocrine looks forward to working closely with the FDA as the review process continues.
Read Announcement- Drug:
- crinecerfont
- Announced Date:
- July 1, 2024
- Indication:
- For the Treatment of Congenital Adrenal Hyperplasia (CAH)
Announcement
Neurocrine Biosciences, Inc. announced the U.S. Food and Drug Administration (FDA) has accepted its two New Drug Applications (NDA) with Priority Review designations for crinecerfont in the treatment of children, adolescents and adults with classic congenital adrenal hyperplasia (CAH).
AI Summary
Neurocrine Biosciences, Inc. announced that the U.S. FDA has accepted its two New Drug Applications (NDAs) for crinecerfont with Priority Review designations. Crinecerfont is a highly selective CRF1 antagonist aimed at treating classic congenital adrenal hyperplasia (CAH) in children, adolescents, and adults. If approved, it would become the first new treatment option for CAH in 70 years. The NDAs include both a capsule formulation and an oral solution, with Prescription Drug User Fee Act (PDUFA) target action dates set for December 29 and December 30, 2024. The Priority Review designation underscores the urgency of providing new therapies for this rare and challenging endocrine disorder, as it recognizes a high unmet medical need. This development represents an important step forward in addressing CAH, offering hope for improved treatment outcomes and a novel, first-in-class approach in managing the disease.
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INGREZZA (Valbenazine) - FDA Regulatory Timeline and Events
INGREZZA (Valbenazine) is a drug developed by Neurocrine Biosciences for the following indication: Tardive Dyskinesia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- INGREZZA (Valbenazine)
- Announced Date:
- June 27, 2025
- Indication:
- Tardive Dyskinesia
Announcement
Neurocrine Biosciences, announced new patient-reported post-hoc data from the KINECT®-HD study highlighting significant reductions in both cognitive and motor-related burdens among adults treated for Huntington's disease chorea with once-daily INGREZZA® (valbenazine) capsules.
AI Summary
Neurocrine Biosciences announced new patient-reported post-hoc data from the Phase 3 KINECT®-HD study. The study highlights that once-daily INGREZZA® (valbenazine) capsules significantly reduced both cognitive and motor-related burdens in adults with Huntington’s disease chorea. Patients noted improvements in areas such as memory loss, decision-making, and difficulty finding the right words, along with enhanced mobility, coordination, and reduced abnormal movements. The analysis used the Huntington’s Disease Health Index to evaluate changes from baseline across various cognitive and motor domains. Overall, the results showed that individuals treated with INGREZZA experienced greater reductions in symptoms compared to those on placebo. These findings suggest that INGREZZA can help manage not only chorea but also additional challenges in cognitive function and motor abilities, offering new hope for patients with Huntington’s disease. The results will be presented at the 2025 ATMRD Congress.
Read Announcement- Drug:
- INGREZZA (Valbenazine)
- Announced Date:
- June 20, 2025
- Indication:
- Tardive Dyskinesia
Announcement
Neurocrine Biosciences, Inc. announced the presentation of new analyses from a Phase 4 randomized withdrawal study demonstrating significant patient-reported improvements in health-related quality of life and functional measures among adults aged 65 years and older with tardive dyskinesia who received continued treatment with INGREZZA® (valbenazine) capsules.
AI Summary
Neurocrine Biosciences, Inc. announced new analyses from a Phase 4 randomized withdrawal study showing that continuous treatment with INGREZZA® (valbenazine) capsules leads to significant improvements in quality of life and functional status for adults aged 65 and older with tardive dyskinesia. The study, which will be presented at the 2025 American Association of Nurse Practitioners National Conference in San Diego, focused on patient-reported outcomes using validated scales to measure health-related quality of life and functional impairment. Patients who continued on INGREZZA reported meaningful benefits compared to those who switched to placebo. These findings add to the growing body of evidence that INGREZZA not only reduces the severity of tardive dyskinesia symptoms but also helps improve overall daily functioning and well-being in an older population, addressing the unique challenges faced by these patients.
Read Announcement- Drug:
- INGREZZA (Valbenazine)
- Announced Date:
- June 2, 2025
- Indication:
- Tardive Dyskinesia
Announcement
Neurocrine Biosciences, Inc. announced new data from the Phase 4 KINECT-PROTM open-label study demonstrating robust and sustained improvements in physical, social and emotional functioning in patients with tardive dyskinesia taking once-daily INGREZZA® (valbenazine) capsules.
AI Summary
Neurocrine Biosciences announced new Phase 4 clinical data from the KINECT-PRO study showing that once-daily INGREZZA® (valbenazine) capsules lead to robust and sustained improvements in patients with tardive dyskinesia. The open-label study used multiple validated patient-reported outcome measures, including the Tardive Dyskinesia Impact Scale, to assess changes in physical, social, and emotional functioning. Results revealed that patients experienced meaningful improvements as early as Week 4, with benefits lasting through Week 24. Notably, even those with milder symptoms at baseline reported a significant reduction in the negative impact of uncontrolled movements on their daily lives. These findings reinforce the potential of INGREZZA to effectively improve quality of life in individuals affected by this challenging condition.
Read Announcement- Drug:
- INGREZZA (Valbenazine)
- Announced Date:
- May 16, 2025
- Indication:
- Tardive Dyskinesia
Announcement
Neurocrine Biosciences, Inc. announced the presentation of new analyses from a Phase 4 randomized withdrawal study (NCT03891862) showing patients with tardive dyskinesia who received continued treatment with INGREZZA® (valbenazine) capsules reported improvements across functional and health-related quality of life measures.
AI Summary
Neurocrine Biosciences, Inc. recently presented new data from a Phase 4 randomized withdrawal study (NCT03891862). The study showed that patients with tardive dyskinesia who continued treatment with INGREZZA® (valbenazine) capsules experienced ongoing improvements in their daily functioning and overall quality of life. These improvements were measured through assessments of mobility, self-care, usual activities, and levels of pain and anxiety, using instruments like the EuroQol 5-Dimension 5-Level (EQ-5D-5L) and the Sheehan Disability Scale (SDS). The results indicate that maintaining INGREZZA® treatment can significantly enhance both functional abilities and health-related quality of life for patients. The findings were presented at the 2025 International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Conference in Montreal, emphasizing the therapy’s potential to positively impact patient care.
Read Announcement- Drug:
- INGREZZA (Valbenazine)
- Announced Date:
- April 24, 2025
- Indication:
- Tardive Dyskinesia
Announcement
Neurocrine Biosciences, Inc. announced publication of a post-hoc analysis from two 48-week studies, the KINECT® 3 extension and KINECT® 4, demonstrating the long-term safety profile and robust efficacy of INGREZZA® (valbenazine) capsules in adults aged 65 years and older with tardive dyskinesia (TD) in The Journal of Clinical Psychiatry.
AI Summary
Neurocrine Biosciences recently published a post-hoc analysis in The Journal of Clinical Psychiatry focusing on the long-term safety and effectiveness of INGREZZA® (valbenazine) capsules for treating tardive dyskinesia (TD) in adults aged 65 and older. The analysis combined data from two 48‐week studies—the KINECT® 3 extension and KINECT® 4—and is the first peer-reviewed examination of a vesicular monoamine transporter 2 inhibitor in this high‐risk population. Results showed that patients 65 and older experienced clinically meaningful improvements in TD symptoms as early as eight weeks into treatment, with benefits sustained through 48 weeks. Importantly, no new treatment-emergent adverse events of clinical concern were observed when compared to younger adults. These findings fill an important research gap and suggest that INGREZZA provides a robust and safe long-term option for managing TD in older adults.
Read Announcement- Drug:
- INGREZZA (Valbenazine)
- Announced Date:
- March 31, 2025
- Indication:
- Tardive Dyskinesia
Announcement
Neurocrine Biosciences, announced the presentation of new data from a real-world study showing that all patients with tardive dyskinesia achieved a therapeutic dose with INGREZZA® (valbenazine) capsules upon initiation of treatment.
AI Summary
Neurocrine Biosciences announced new data from a real-world study presented at the Academy of Managed Care Pharmacy 2025 Annual Meeting. The study focused on the use of INGREZZA® (valbenazine) capsules in patients with tardive dyskinesia. The key finding was that every patient reached a therapeutic dose on the first day of treatment with INGREZZA®, eliminating the need for gradual titration. This result suggests that INGREZZA® offers a more efficient treatment start compared to other VMAT2 inhibitors.
In contrast, only about half of the patients taking deutetrabenazine were able to reach a therapeutic dose within six months. This difference could lessen the clinical and patient burden related to dosing adjustments, making INGREZZA® a promising option for quickly managing the symptoms of tardive dyskinesia.
Read Announcement- Drug:
- INGREZZA (Valbenazine)
- Announced Date:
- March 20, 2025
- Indication:
- Tardive Dyskinesia
Announcement
Neurocrine Biosciences, presented new data from the long-term, open-label KINECT® 4 study demonstrating remission of tardive dyskinesia among the majority of patients treated with once-daily INGREZZA® (valbenazine) capsules. This analysis was presented at the 2025 Psychiatry Update Conference in Chicago.
AI Summary
Neurocrine Biosciences presented promising results from the long-term KINECT 4 study at the 2025 Psychiatry Update Conference in Chicago. The study showed that a majority of patients with tardive dyskinesia achieved remission after 48 weeks of treatment with once-daily INGREZZA (valbenazine). Regardless of whether participants received a 40 mg or 80 mg dosage, about 59% met the remission threshold based on the Abnormal Involuntary Movement Scale. This threshold was defined by minimal or no movement issues in the seven key body regions. Importantly, these results were consistent across patients with different psychiatric conditions, including schizophrenia, schizoaffective disorder, and mood disorders. The findings reinforce INGREZZA’s effectiveness as a long-term treatment option for managing tardive dyskinesia and offer new hope for patients in need of improved movement control and quality of life.
Read Announcement- Drug:
- INGREZZA (Valbenazine)
- Announced Date:
- March 17, 2025
- Indication:
- Tardive Dyskinesia
Announcement
Neurocrine Biosciences, Inc. presented data from the KINECT®-HD study showcasing significant improvements in chorea across body regions with once-daily INGREZZA® (valbenazine) capsules in adults with Huntington's disease.
AI Summary
Neurocrine Biosciences recently showcased encouraging findings from the Phase 3 KINECT®-HD study at a neuroscience symposium. The study focused on using once-daily INGREZZA® (valbenazine) capsules to treat chorea in adults with Huntington’s disease. In a post-hoc analysis, researchers found that INGREZZA significantly reduced involuntary movements across several body regions. Improvements were most notable in the arms and legs, which exhibited the highest level of chorea severity at baseline. These results were measured using the Unified Huntington’s Disease Rating Scale (UHDRS®) Total Maximal Chorea (TMC) score, highlighting a meaningful benefit for patients. The data suggest that INGREZZA offers consistent relief from chorea symptoms, potentially improving everyday functioning for those affected. The study’s findings support INGREZZA’s role in addressing the complex motor challenges associated with Huntington’s disease.
Read Announcement- Drug:
- INGREZZA (Valbenazine)
- Announced Date:
- September 30, 2024
- Indication:
- Tardive Dyskinesia
Announcement
Neurocrine Biosciences, announced interim results from the ongoing open-label KINECT®-HD2 study of INGREZZA® (valbenazine) capsules for the long-term treatment of adults with chorea associated with Huntington's disease.
AI Summary
Neurocrine Biosciences has announced interim results from its ongoing open‐label KINECT®-HD2 study, which evaluates the long-term use of INGREZZA® (valbenazine) capsules in adults with Huntington’s disease chorea. The study showed that significant improvements in chorea symptoms were observed as early as Week 2 and sustained up to Week 104. Improvement was measured through changes in the Unified Huntington’s Disease Rating Scale Total Maximal Chorea score, with both patients and clinicians reporting that over 70% experienced “much improved” or “very much improved” symptoms. Importantly, these positive results were consistent even among patients who were using antipsychotic medications concurrently. This interim analysis underscores the long-term safety and effectiveness of INGREZZA in managing the disruptive motor symptoms seen in Huntington’s disease, providing valuable hope for sustained treatment benefits in this challenging condition.
Read Announcement- Drug:
- INGREZZA (Valbenazine)
- Announced Date:
- May 21, 2024
- Indication:
- Tardive Dyskinesia
Announcement
Neurocrine Biosciences, announced the publication of data from a post hoc analysis of the Phase 3 KINECT®-4 study of INGREZZA® (valbenazine) capsules in the Journal of Clinical Psychopharmacology.
AI Summary
Neurocrine Biosciences recently announced the publication of data from a post hoc analysis of its Phase 3 KINECT®-4 study in the Journal of Clinical Psychopharmacology. This analysis focused on the long-term benefits of INGREZZA® (valbenazine) capsules in the real-world management of tardive dyskinesia (TD). The study showed that nearly all participants reached a clinically meaningful improvement in TD symptom severity by Week 48. This finding is significant because TD is a persistent condition that often requires continuous treatment to sustain its benefits. The results confirm that a once-daily capsule of INGREZZA provides both reliable long-term efficacy and tolerability, making it a valuable treatment option in clinical practice. These encouraging findings further support the use of INGREZZA for managing TD symptoms over an extended period.
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NBI-1065845 - FDA Regulatory Timeline and Events
NBI-1065845 is a drug developed by Neurocrine Biosciences for the following indication: In Adults with Major Depressive Disorder.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- NBI-1065845
- Announced Date:
- January 28, 2025
- Indication:
- In Adults with Major Depressive Disorder
Announcement
Neurocrine Biosciences, announced the initiation of a Phase 3 registrational study to evaluate the efficacy, safety and tolerability of osavampator (formerly NBI-1065845), an investigational drug under development as an adjunctive treatment to antidepressants for major depressive disorder (MDD).
AI Summary
Neurocrine Biosciences announced the start of a Phase 3 registrational study to test osavampator as an additional treatment to standard antidepressants in adults with major depressive disorder (MDD). The study aims to evaluate the drug’s safety, effectiveness, and tolerability in patients who have not responded well to current treatments. Previous Phase 2 data from the SAVITRI study showed that osavampator met both its primary and secondary endpoints and was generally well tolerated, strengthening hope that this new treatment could help the millions suffering from MDD.
This new Phase 3 trial will focus on participants with a primary diagnosis of MDD and an inadequate response to typical antidepressants. Researchers believe osavampator has the potential to be a first-in-class treatment that could offer a new strategy for patients where current options fall short.
Read Announcement- Drug:
- NBI-1065845
- Announced Date:
- April 23, 2024
- Indication:
- In Adults with Major Depressive Disorder
Announcement
Neurocrine Biosciences announced positive topline data for its Phase 2 SAVITRI™ study.
AI Summary
Neurocrine Biosciences announced that its Phase 2 SAVITRI™ study met its primary endpoint, showing a statistically significant reduction in depression symptoms as measured by the Montgomery Åsberg Depression Rating Scale (MADRS) at Day 28. One dose of NBI‐1065845 led to an improvement of -4.3 points compared to placebo, while the improvement increased to -7.5 points at Day 56 for the same dose. The study also met key secondary endpoints with significant improvements continuing through Day 56. Additionally, NBI‐1065845 was generally well-tolerated, with headache being the most common side effect and a safety profile similar to placebo. Researchers are encouraged by these results as they suggest that NBI‐1065845 has potential as a new treatment option for adults with major depressive disorder who have not fully benefited from other antidepressants.
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NBI-1117567 - FDA Regulatory Timeline and Events
NBI-1117567 is a drug developed by Neurocrine Biosciences for the following indication: for the potential treatment of neurological and neuropsychiatric conditions.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- NBI-1117567
- Announced Date:
- May 8, 2024
- Indication:
- for the potential treatment of neurological and neuropsychiatric conditions.
Announcement
Neurocrine Biosciences, Inc. announced the initiation of its Phase 1 first-in-human clinical study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of investigational compound NBI-1117567 in healthy adult participants.
AI Summary
Neurocrine Biosciences, Inc. recently announced the start of its Phase 1, first‐in‐human clinical study. This early-phase trial is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the investigational compound NBI-1117567. The study will involve healthy adult participants and aims to determine how the compound is absorbed, distributed, metabolized, and excreted, as well as the overall effect it has on the body.
The trial is an important first step in understanding the potential therapeutic benefits of NBI-1117567 and any possible risks associated with its use. Results from this study will help guide future research and the compound’s further development. By successfully completing these initial evaluations, Neurocrine Biosciences hopes to lay the groundwork for additional clinical trials in the coming years.
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NBI-1117568 - FDA Regulatory Timeline and Events
NBI-1117568 is a drug developed by Neurocrine Biosciences for the following indication: For the treatment of schizophrenia and other neuropsychiatric disorders.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- NBI-1117568
- Announced Date:
- May 28, 2025
- Indication:
- For the treatment of schizophrenia and other neuropsychiatric disorders
Announcement
Neurocrine Biosciences, Inc. announced the presentation of data from the Phase 2 study of NBI-1117568 in adults with schizophrenia, which showed a significant improvement in symptoms and overall severity and highlighted new data on the safety and tolerability of the treatment.
AI Summary
Neurocrine Biosciences, Inc. recently presented data from its Phase 2 study on NBI-1117568, an investigational oral muscarinic M4 selective orthosteric agonist being developed for schizophrenia. The study showed that treatment with 20 mg once daily significantly improved schizophrenia symptoms and the overall severity of the disease compared to placebo, with noticeable changes as early as Week 3. Safety and tolerability data from the trial were also highlighted, with adverse events such as somnolence and dizziness reported at slightly higher rates than placebo, though discontinuation rates remained similar between the groups.
These positive findings, shared at the American Society of Clinical Psychopharmacology 2025 Annual Meeting, support further evaluation of NBI-1117568. Based on the favorable results, Neurocrine is advancing this compound into a Phase 3 registrational program to rigorously assess its long-term efficacy and safety in adults with schizophrenia.
Read Announcement- Drug:
- NBI-1117568
- Announced Date:
- April 30, 2025
- Indication:
- For the treatment of schizophrenia and other neuropsychiatric disorders
Announcement
Neurocrine Biosciences, Inc. announced the initiation of a Phase 3 registrational program to evaluate the efficacy, safety and tolerability of NBI-1117568, the company's investigational oral muscarinic M4 selective orthosteric agonist, as a potential treatment for schizophrenia.
AI Summary
Neurocrine Biosciences announced the start of a Phase 3 registrational program to evaluate NBI-1117568, an investigational oral muscarinic M4 selective orthosteric agonist for treating schizophrenia. This global, double-blind, placebo-controlled study will enroll around 280 adult patients who are experiencing an acute worsening or relapse of symptoms. The trial aims to measure improvements in symptoms using the Positive and Negative Syndrome Scale (PANSS) as the primary endpoint and the Clinical Global Impression of Severity (CGI-S) scale as a key secondary endpoint.
Supported by positive Phase 2 results where the once-daily 20 mg dose notably reduced schizophrenia symptoms, the Phase 3 study is designed to further assess the drug’s efficacy, safety, and tolerability. Neurocrine Biosciences hopes this study will offer a new, promising treatment option for people living with schizophrenia.
Read Announcement- Drug:
- NBI-1117568
- Announced Date:
- August 28, 2024
- Indication:
- For the treatment of schizophrenia and other neuropsychiatric disorders
Announcement
Neurocrine Biosciences, Inc. announced positive top-line data for its Phase 2 clinical study of NBI-1117568 (NBI-'568) in adults with schizophrenia. NBI-'568 is the first investigational, oral, muscarinic M4 selective agonist in development for the treatment of schizophrenia.
AI Summary
Neurocrine Biosciences announced positive top-line results from a Phase 2 study in adults with schizophrenia for its investigational drug NBI-1117568. This once-daily, 20 mg oral compound—being the first muscarinic M4 selective agonist in development—showed a statistically significant 7.5-point improvement on the PANSS total score compared to placebo at Week 6. The study also reported a meaningful improvement of 18.2 points from baseline along with significant enhancements in secondary measures such as the Clinical Global Impression of Severity Scale and Marder Factor Scores for both positive and negative symptoms.
The Phase 2 trial demonstrated that NBI-1117568 was generally safe and well tolerated, with similar rates of adverse events to placebo. Based on these promising results, Neurocrine plans to advance the drug into Phase 3 clinical development in early 2025 as a potential new therapy option for schizophrenia.
Read Announcement- Drug:
- NBI-1117568
- Announced Date:
- April 16, 2024
- Indication:
- For the treatment of schizophrenia and other neuropsychiatric disorders
Announcement
Nxera Pharma Co., Ltd formerly known as Sosei Group or Sosei Heptares – has been notified by its partner Neurocrine Biosciences Inc that NBI-1117568, an oral selective muscarinic M4 receptor agonist being advanced in Phase 2 clinical trials by Neurocrine for the treatment of schizophrenia and other neuropsychiatric disorders, has successfully completed a long-term preclinical toxicity program that meets US FDA requirements to allow for safe, chronic (i.e. long-term) dosing in future clinical trials. .
AI Summary
Nxera Pharma, formerly known as Sosei Heptares, announced that its partner Neurocrine Biosciences has confirmed the successful completion of a long-term preclinical toxicity program for NBI-1117568. This oral, selective muscarinic M4 receptor agonist is being advanced into Phase 2 clinical trials for schizophrenia and other neuropsychiatric disorders. The study met US FDA requirements for safe, chronic dosing, marking an important safety milestone.
This achievement triggers a $15 million payment to Nxera from Neurocrine under their collaboration agreement. The results support the continued development of NBI-1117568, showing promise for its long-term use in treating neurological conditions where patients require durable therapy. This success underlines the productive partnership between Nxera and Neurocrine, which is working together to bring innovative treatments to patients with unmet medical needs.
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NBI-1140675 - FDA Regulatory Timeline and Events
NBI-1140675 is a drug developed by Neurocrine Biosciences for the following indication: VMAT2 Inhibitor.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- NBI-1140675
- Announced Date:
- March 5, 2025
- Indication:
- VMAT2 Inhibitor
Announcement
Neurocrine Biosciences, Inc. announced the initiation of a Phase 1 clinical study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of investigational compound NBI-1140675 in healthy adult participants.
AI Summary
Neurocrine Biosciences, Inc. has begun a Phase 1 clinical study to test the investigational compound NBI‑1140675 in healthy adult participants. This study will evaluate the drug’s safety, tolerability, pharmacokinetics, and pharmacodynamics. NBI‑1140675 is an oral, selective second‑generation inhibitor of the vesicular monoamine transporter 2 (VMAT2) and is being developed to potentially treat certain neurological and neuropsychiatric conditions.
The trial is designed to understand how the compound behaves in the body and to determine its proper dosage levels for future studies. The research is part of Neurocrine’s ongoing efforts to extend its portfolio of VMAT2 inhibitors and develop treatments that may offer differentiated benefits for patients with these challenging conditions.
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Valbenazine (KINECT-HD) - FDA Regulatory Timeline and Events
Valbenazine (KINECT-HD) is a drug developed by Neurocrine Biosciences for the following indication: Chorea in Huntington Disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Valbenazine (KINECT-HD)
- Announced Date:
- July 18, 2024
- Indication:
- Chorea in Huntington Disease
Announcement
Neurocrine Biosciences, announced the launch of a new sprinkle formulation of INGREZZA® (valbenazine) capsules, the #1 prescribed vesicular monoamine transporter 2 (VMAT2) inhibitor indicated for the treatment of adults with tardive dyskinesia and chorea associated with Huntington's disease.1
AI Summary
Neurocrine Biosciences has launched a new sprinkle formulation of INGREZZA® (valbenazine) capsules. This innovative version is designed for adults suffering from tardive dyskinesia or chorea associated with Huntington's disease who have difficulty swallowing pills. The sprinkle formulation contains the same active ingredient and comes in easy-to-administer options of 40 mg, 60 mg, and 80 mg. Patients or caregivers can open the capsule and sprinkle the granules on soft food like applesauce, yogurt, or pudding, making daily administration simpler.
INGREZZA SPRINKLE offers the same therapeutic benefits as the original capsule, providing a full effective dose from day one with one capsule once daily. It is available through the familiar network of specialty and local pharmacies, ensuring that patients continue to receive consistent treatment without the challenge of swallowing whole capsules.
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