Drugmaker with rare disease work · based in San Diego, United States · founded 1992 · ticker NBIX · neurocrine.com
Neurocrine Biosciences works on 6 rare diseases we track, including Congenital Adrenal Hyperplasia, Friedreich Ataxia and 4 more. It has 4 active clinical trials, and 1 is looking for volunteers now. It also has 3 FDA-approved rare disease drugs.
Neurocrine Biosciences develops treatments for rare and under-addressed neurological, neuroendocrine and neuropsychiatric disorders. The company markets INGREZZA (valbenazine) for tardive dyskinesia and Huntington's chorea, CRENESSITY (crinecerfont), approved in December 2024 as a first-in-class, non-steroidal add-on treatment for classic congenital adrenal hyperplasia, and VYKAT XR (diazoxide choline), the first FDA-approved medicine for hyperphagia in Prader-Willi syndrome, added through the $2.9 billion acquisition of Soleno Therapeutics that closed in May 2026.
Neurocrine Biosciences pipeline
Each row lists the diseases Neurocrine Biosciences is studying at that stage, from the ones closest to approval down to the earliest work.
These are Neurocrine Biosciences trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
1 trial looking for volunteers now
Looking for volunteersPhase 2Recently updatedNCT07536269
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old
The main objective of this study is to assess the safety and tolerability of crinecerfont in pediatric participants 3 months to \<4 years of age with CAH.
The main objective for this study is to evaluate the pharmacokinetics (PK) of crinecerfont in pediatric participants 0 to \<2 years of age with congenital adrenal hyperplasia (CAH).
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).
Underway, not taking new volunteersPhase 3Hasn't posted an update in over a yearNCT04806451
Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)
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).
Follow a condition Neurocrine Biosciences is studying
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Trial Alerts, when a new trial opens or a trial changes
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Friedreich ataxia is an autosomal recessiveneurodegenerative disease causing progressive damage to the nervous system, resulting in loss of coordination (ataxia), weakness, and heart problems.
Huntington disease is an autosomal dominantneurodegenerative disorder caused by an expanded CAG trinucleotide repeat in the huntingtin gene.
How common: About 3-7 per 100,000 people of European ancestry, according to MedlinePlus Genetics; less common in people of Japanese, Chinese and African descent
Multiple sclerosis is a chronicautoimmune disease where the immune system attacks the protective myelin sheath surrounding nerve fibers in the brain and spinal cord.
How common: Approximately 1 million people in the U.S.; about 1 in 300
Prader-Willi Syndrome is a rare genetic disorder affecting chromosome 15 that results in weak muscle tone, low hormone levels, incomplete sexual development, intellectual disability, and a constant...
How common: Approximately 1 in 10,000 to 30,000 live births
SCN8Aencephalopathy is a rare genetic epilepsy caused by a change in the SCN8A gene, which builds the Nav1.6 sodium channel that brain cells use to fire.
How common: Unknown; estimated to account for about 1% of epilepsies with encephalopathy (MedlinePlus Genetics), and GeneReviews states the true prevalence has not been established
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Neurocrine Biosciences's own site is neurocrine.com, and our help paying for treatment page lists drugmaker programs and charity funds.
Neurocrine Biosciences develops treatments for rare and under-addressed neurological, neuroendocrine and neuropsychiatric disorders. The company markets INGREZZA (valbenazine) for tardive dyskinesia and Huntington's chorea, CRENESSITY (crinecerfont), approved in December 2024 as a first-in-class, non-steroidal add-on treatment for classic congenital adrenal hyperplasia, and VYKAT XR (diazoxide choline), the first FDA-approved medicine for hyperphagia in Prader-Willi syndrome, added through the $2.9 billion acquisition of Soleno Therapeutics that closed in May 2026.
Neurocrine Biosciences has active programs across 6 rare diseases: Congenital Adrenal Hyperplasia, Friedreich Ataxia, Huntington Disease, Multiple Sclerosis, Prader-Willi Syndrome, SCN8AEncephalopathy. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.
Neurocrine Biosciences is a diversified pharmaceutical company headquartered in San Diego, United States, founded in 1992. While it operates across multiple therapeutic areas, it maintains a significant rare disease portfolio with 6 active rare disease programs including Congenital Adrenal Hyperplasia, Friedreich Ataxia, Huntington Disease. Many of its rare disease treatments hold orphan drug designations from the FDA.
The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Neurocrine Biosciences, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.
To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.
Neurocrine Biosciences has 3 FDA-approved drugs with a rare disease use that we track, as of November 2026.
Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.
Yes, for 3 of its FDA-approved rare disease medicines that we track, each checked on Neurocrine Biosciences' own website.
Crenessity: Neurocrine Access Support, 1-855-276-7489. Offers copay help with private insurance, a free medicine program and insurance help. Checked October 26, 2026.
Ingrezza: Neurocrine Access Support, 1-844-647-3992. Offers copay help with private insurance and a free medicine program. Checked October 12, 2026.
Vykat XR: Soleno One, 1-833-765-3661. Offers copay help with private insurance and insurance help. Checked October 19, 2026.
Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page.
Eligibility is decided by each program, so call the number listed before a prescription is filled.
Neurocrine Biosciences was founded in 1992 and is headquartered in San Diego, United States. The company has been active for 34 years. It trades publicly under the ticker symbol NBIX.