Applying
Brave

Science to Diseases
and Conditions

We have an FDA-approved treatment for tardive dyskinesia and Huntington’s disease chorea, as well as a robust pipeline including multiple compounds in mid-to-late phase clinical development across our core therapeutic areas.
 
 

 
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While others walk away from complex disease states, our team
persists.
Neurocrine Biosciences is fully invested in bringing medicines to market for people with under-addressed and rare diseases.

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  • EE-CSWS typical onset occurs between the ages of two and four years old, with seizures occurring primarily during sleep. The seizures interfere with processes critical to learning and memory. Diagnosis of EE-CSWS is based on a unique electroencephalogram (EEG) pattern of electrical status epilepticus during sleep (ESES), together with cognitive stagnation and regression. Following puberty, seizure frequency tends to decline, however, developmental delays often remain.

    EE-CSWS impacts less than 2% of children living with epilepsy worldwide. There is currently no approved treatment for the disorder.

    See What We Are Working On >

    SCN8A-DEE causes a range of symptoms including severe epilepsy, early onset developmental delay, cognitive impairment, and other medical challenges. Typical onset occurs around 4 months of age.

    There are no approved therapies for this form of pediatric epilepsy.

    See What We Are Working On >

  • Congenital Adrenal Hyperplasia (CAH)

    CAH is a rare genetic condition that results in an enzyme deficiency (~95% due to 21-hydroxylase [21-OHD] deficiency) that alters the production of adrenal hormones.

    CAH is a is a rare genetic condition that affects the adrenal glands. The adrenal glands produce hormones that are essential to life. In about 95% of CAH cases, a genetic change (mutation) results in the deficiency, or lack, of the enzyme 21-hydroxylase (21-OHD). 21-OHD is needed for the adrenal glands to make two important hormones: cortisol and aldosterone. Cortisol allows the body to respond to injury, stress, or illness. Aldosterone helps regulate blood pressure and salt levels. A severe deficiency of 21-OHD stops the adrenal glands from making cortisol, and in about 75% of these cases, it also affects aldosterone production. This type of severe deficiency is estimated to affect up to approximately 30,000 people in the US and 50,000 people in Europe. If left untreated, CAH can result in excessive salt loss, dehydration, and even death.

    In CAH, the body doesn’t make enough cortisol and makes too many adrenal androgens. This happens in a biological system within the body called the hypothalamus-pituitary-adrenal (HPA) axis. When cortisol is low, a signal is sent to the hypothalamus to release a hormone called corticotropin-releasing factor (CRF). CRF binds to corticotropin-releasing factor type 1 (CRF1) receptors in the pituitary gland to signal the release of adrenocorticotropic hormone (ACTH). ACTH then signals the adrenal glands to produce androgens, aldosterone, and cortisol. When there is enough cortisol in the body, the hypothalamus stops releasing CRF. But in untreated or undertreated CAH, there isn’t enough cortisol. That means the body keeps making CRF, leading to an increase in CRF1 receptor activity and, therefore, ACTH. Then the high levels of ACTH cause the adrenal glands to produce too many androgens.

    The only treatment currently approved by the US Food and Drug Administration for CAH is glucocorticoids (GCs). These are commonly called steroids. GCs are used to treat cortisol deficiency and to help decrease the production of androgens. However, greater-than-normal (supraphysiological) GC doses are often needed to reduce the high levels of CRF and ACTH that result in adrenal androgen excess. Taking these higher doses of GCs long term can lead to serious side effects, including weight gain, diabetes, cardiovascular disease and osteoporosis. They can also affect mental health and cognition, causing changes in mood and memory. On the other hand, taking lower GC doses can result in androgen excess. This can lead to growth and development problems in pediatric patients, female health problems such as acne, excess hair growth and menstrual irregularities, testicular rest tumors in males and fertility issues in both sexes.

  • As one of the leading causes of disability worldwide, schizophrenia often results in significant emotional burden for those who experience symptoms, as well as their family and friends. It impacts approximately 24 million people worldwide. Approximately 33% of patients with schizophrenia fail to respond to current antipsychotic therapy.

    See What We Are Working On >

    Carlos’ Journey Living with Schizophrenia

    Hear more of Carlos’ Story

    MDD is characterized by a persistently depressed mood, loss of interest, lack of enjoyment in daily activities, and decreased energy that can impact normal daily functioning, relationships, and overall quality of life. Of the more than 16 million people in the U.S. who live with MDD, about one-third do not respond to available antidepressants.

    See What We Are Working On >

  • Symptoms of endometriosis include painful periods, pelvic pain between periods, and pain with sex. Estrogen fuels the growth of lesions that can occur on the ovaries, the fallopian tubes, or other areas near the uterus, such as the bowel or bladder.

    According to researchers, 7.5 million women in the U.S. are diagnosed with endometriosis, with 3 million diagnosed with moderate to severe endometriosis.

    See What We’ve Developed* >

    *All commercialization and marketing by AbbVie Inc.

    Uterine fibroids are most often seen in women between ages 30 to 40 but can occur at any age. Uterine fibroids are the most common pelvic growth, affecting around 20% of all women by age 59. Uterine fibroids are the leading cause of infertility.

    Some common symptoms associated with uterine fibroids include heavy menstrual bleeding, painful periods, vaginal bleeding at times other than menstruation, anemia, pain in the abdomen or lower back, pain during sex, difficulty urinating or frequent urination, constipation, rectal pain, or difficulty getting pregnant.

    See What We’ve Developed* >

    *All commercialization and marketing by AbbVie Inc.

References

1. University of Michigan Health. Movement disorders. Accessed July 12, 2022. https://www.uofmhealth.org/conditions-treatments/brain-neurological-conditions/movement-disorders
2. MedlinePlus. Movement disorders. Accessed July 12, 2022. https://medlineplus.gov/movementdisorders.html
3. Epilepsy Foundation. Who can get epilepsy? Accessed July 12, 2022. https://www.epilepsy.com/learn/about-epilepsy-basics/who-gets-epilepsy
4. National Organization for Rare Disorders. Congenital adrenal hyperplasia. Accessed July 12, 2022. https://rarediseases.org/rare-diseases/congenital-adrenal-hyperplasia/
5. Our World in Data. Mental health. Accessed July 12, 2022. https://ourworldindata.org/mental-health
6. National Library of Medicine. MedlinePlus. Uterine fibroids. Accessed July 12, 2022. https://medlineplus.gov/uterinefibroids.html#
7. National Library of Medicine. MedlinePlus. Endometriosis. Accessed July 12, 2022. https://medlineplus.gov/endometriosis.html

Learn more about SCN8A from patients and healthcare professionals

From new parents to an SCN8A diagnosis

A good day for Elliott. A bad day for Elliot.

A diagnosis for the entire family

The search for answers, hope, and a future for all SCN8A kids.

Unremarkable pregnancy to an SCN8A diagnosis

Caring for Stella and their hopes for the future

Hear from Dr. Schreiber on SCN8A History, Early Diagnosis and Hope for Future Medication

Learn more about CAH from patients and healthcare providers

Madison’s story

CAH Diagnosis and Life Challenges

Living with CAH

Achieving Androgen Control in CAH

CAH Treatment Challenges

Carlos’ Journey Living with Schizophrenia

Carlos’ Journey Living with Schizophrenia

Hear more of Carlos’ Story

Hear more of Carlos’ Story

Learn more about CAH from patients, caregivers, and healthcare providers

Madison’s story

CAH Diagnosis and Life Challenges

Living with CAH

Achieving Androgen Control in CAH

CAH Treatment Challenges

A Mom’s Story: Learning about CAH

A Mom’s Story: Living With CAH

A Mom’s Story: Monitoring CAH

A Mom’s Story: Medications and Physician Visits

A Mom's Story: Adolescence and the Future

A Patient’s Story: Learning about CAH

A Patient’s Story: Growing Up With CAH

A Patient’s Story: Managing My CAH

A Patient’s Story: Frustrations and Hope for the Future