Otsuka Pharmaceutical
Otsuka Pharmaceutical works on 9 rare diseases tracked on Trial Friend, including Focal Segmental Glomerulosclerosis, Geographic Atrophy, IgA Nephropathy and 6 more, with 15 recruiting clinical trials and 4 FDA-approved rare disease drugs.
Otsuka Pharmaceutical develops treatments for rare genetic and renal diseases. Key products include JYNARQUE (tolvaptan), the first treatment to slow kidney function decline in autosomal dominant polycystic kidney disease (approved 2018), and Voyxact (sibeprenlimab), approved in November 2025 for IgA nephropathy. The company also has repinatrabit in Phase 3 for phenylketonuria (initiated December 2025).
Focus areas at Otsuka Pharmaceutical
Within its broader pharmaceutical portfolio, Otsuka Pharmaceutical has active clinical trial programs and drug development efforts across 9 rare diseases, including Focal Segmental Glomerulosclerosis, Geographic Atrophy, IgA Nephropathy, IgA Vasculitis with Nephritis, Myelodysplastic Syndromes, and 4 additional rare conditions. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows all active and recruiting studies sponsored by Otsuka Pharmaceutical, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Otsuka Pharmaceutical is headquartered in Tokyo, Japan, founded in 1964, publicly traded under the ticker symbol 4578. The company maintains a dedicated rare disease division alongside its broader therapeutic portfolio, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Otsuka Pharmaceutical Drug Pipeline
Otsuka Pharmaceutical has 15 active clinical trials across 3 development stages, with 15 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 includes all of Otsuka Pharmaceutical's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
Otsuka Pharmaceutical Clinical Trials (15)
Active and recruiting clinical trials sponsored by Otsuka Pharmaceutical, 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.
Otsuka Pharmaceutical FDA-Approved Drugs (4)
Medications developed or marketed by Otsuka Pharmaceutical 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 |
|---|---|---|---|
| ARIPIPRAZOLE Atypical Antipsychotic [EPC] | Abilify Asimtufii intramuscular | Apr 27, 2023 | |
| TOLVAPTAN Vasopressin V2 Receptor Antagonist [EPC] | Jynarque oral | Apr 23, 2018 | |
| BREXPIPRAZOLE Atypical Antipsychotic [EPC] | Rexulti oral | Jul 10, 2015 | |
| SIBEPRENLIMAB | VOYXACT subcutaneous | Nov 25, 2025 |
Otsuka Pharmaceutical Trial Locations
Otsuka Pharmaceutical clinical trials are running at 434 sites in 35 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 (9)
Diseases targeted by Otsuka Pharmaceutical's clinical trial and drug development programs
Focal Segmental Glomerulosclerosis (FSGS) is a kidney disease characterized by scarring of some glomeruli, the tiny filtering units that remove waste from the blood. The disease can develop on its own...
Geographic atrophy is the advanced form of dry age-related macular degeneration, characterized by progressive loss of retinal pigment epithelium, photoreceptors, and choriocapillaris in well-defined a...
IgA nephropathy is the most common primary glomerulonephritis worldwide, caused by abnormal IgA1 antibodies depositing in the kidney's mesangium, activating complement pathways and triggering inflamma...
IgA vasculitis is a small-vessel vasculitis characterized by IgA immune complex deposition in kidneys and other organs. The condition manifests with purpura, arthritis, and glomerulonephritis. Most pa...
Myelodysplastic syndromes are a group of blood cancers in which the bone marrow fails to produce enough healthy blood cells. Immature blood cells do not develop properly and either die in the bone mar...
Phenylketonuria is an inherited metabolic disorder caused by a missing enzyme that prevents the body from properly breaking down the amino acid phenylalanine. This causes a buildup that can damage the...
Patient Resources
Organizations and resources related to Otsuka Pharmaceutical's rare disease focus areas
Frequently Asked Questions About Otsuka Pharmaceutical
Common questions about Otsuka Pharmaceutical's rare disease programs, clinical trials, and treatments.