Otsuka Pharmaceutical

A Trial in Healthy Adult Participants and Adults With Autoimmune Diseases to Test How HBM7020 is Tolerated and How it Behaves in the Body

Tests HBM7020

For Rheumatoid Arthritis, Systemic Lupus Erythematosus, Systemic Sclerosis and Sjögren's Disease

Evaluation of the Efficacy and Safety of EB-1020 in Adult ADHD Patients

Tests EB-1020 (Centanafadine) 164.4 mg, EB-1020 (Centanafadine) 328.8 mg, Placebo

For Attention Deficit Hyperactivity Disorder (ADHD)

A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Tests Tolvaptan Suspension, Tolvaptan Tablets

For Autosomal Recessive Polycystic Kidney (ARPKD)

A Long-term Trial of EB-1020 in Adult Patients With ADHD

Tests EB-1020 (Centanafadine) 164.4 mg, EB-1020 (Centanafadine) 328.8 mg

For Attention Deficit Hyperactivity Disorder (ADHD)

A Study of JNT-517 in Participants With Phenylketonuria (PKU)

Tests JNT-517, JNT-517 Matching Placebo

For Phenylketonuria

LUPKYNIS Drug-use Results Survey

Tests Voclosporin (LUPKYNIS)

For Lupus Nephritis

A Trial to Examine if Repinatrabit is Processed Differently in Adults With Reduced Liver or Kidney Function Compared to Adults With Normal Liver and Kidney Function

Tests Repinatrabit

For Phenylketonuria

A Long-Term Study of JNT-517 in Participants With Phenylketonuria

Tests JNT-517

For Phenylketonuria (PKU)

Otsuka Pharmaceutical is a Japanese drugmaker whose medicines span mental health, kidney disease and rare disease. Its rare disease medicines include Jynarque (tolvaptan), approved in April 2018 as the first treatment to slow kidney function decline in adults at risk of rapidly progressing autosomal dominant polycystic kidney disease, and Voyxact (sibeprenlimab), given accelerated approval on November 25, 2025 to lower protein in the urine of adults with primary IgA nephropathy at risk of getting worse. On October 22, 2026 Otsuka said it had completed its application to the FDA for traditional approval of Voyxact, using 2-year kidney function results from the VISIONARY trial. Otsuka is also testing repinatrabit (code name JNT-517) in Phase 3 for phenylketonuria, with the main trial started in October 2025, and holds an exclusive global license from Ionis for ulefnersen in ALS caused by FUS gene changes, whose Phase 3 FUSION trial met its main goal in September 2026.

Otsuka Pharmaceutical has active programs across 11 rare diseases: Acute Myeloid Leukemia, Amyotrophic Lateral Sclerosis, Focal Segmental Glomerulosclerosis, IgA Nephropathy, Lupus Nephritis, Membranous Nephropathy, Myelodysplastic Syndromes, Phenylketonuria, Polycystic Kidney Disease, Sjögren's Syndrome, Systemic Sclerosis. These span several therapeutic areas. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

Otsuka Pharmaceutical is a diversified pharmaceutical company headquartered in Tokyo, Japan, founded in 1964. While it operates across multiple therapeutic areas, it maintains a significant rare disease portfolio with 11 active rare disease programs including Acute Myeloid Leukemia, Amyotrophic Lateral Sclerosis, Focal Segmental Glomerulosclerosis. 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 Otsuka Pharmaceutical, 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.

Otsuka Pharmaceutical was founded in 1964 and is headquartered in Tokyo, Japan. The company has been active for 62 years. It trades publicly under the ticker symbol 4578.