Neurocrine Biosciences
Neurocrine Biosciences works on 4 rare diseases tracked on Trial Friend, including Congenital Adrenal Hyperplasia, Friedreich Ataxia, Huntington Disease and 1 more, with 4 active clinical trials (1 recruiting) and 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 Drug Pipeline
Neurocrine Biosciences has 4 active clinical trials across 2 development stages, with 1 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Neurocrine Biosciences Clinical Trials (4)
Active and recruiting clinical trials sponsored by Neurocrine Biosciences, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
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Neurocrine Biosciences FDA-Approved Drugs (3)
Medications developed or marketed by Neurocrine Biosciences that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| CRINECERFONT | CRENESSITY | Dec 13, 2024 | |
| VALBENAZINE | INGREZZA oral | Apr 11, 2017 | |
| DIAZOXIDE CHOLINE | VYKAT XR | Mar 26, 2025 |
Neurocrine Biosciences Trial Locations
Neurocrine Biosciences clinical trials are running at 130 sites in 18 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (4)
Diseases targeted by Neurocrine Biosciences's clinical trial and drug development programs
Congenital adrenal hyperplasia comprises rare autosomal recessive disorders of cortisol synthesis. Over 90% result from 21-hydroxylase deficiency. Salt-wasting and virilizing forms present with differ...
Friedreich ataxia is an autosomal recessive neurodegenerative disease causing progressive damage to the nervous system, resulting in loss of coordination (ataxia), weakness, and heart problems. The co...
Huntington disease is an autosomal dominant neurodegenerative disorder caused by an expanded CAG trinucleotide repeat in the huntingtin gene. The progressive disease causes movement problems, cognitiv...
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 fe...
Patient Resources
Organizations and resources related to Neurocrine Biosciences's rare disease focus areas
Frequently Asked Questions About Neurocrine Biosciences
Common questions about Neurocrine Biosciences's rare disease programs, clinical trials, and treatments.