Otsuka Pharmaceutical works on 11 rare diseases tracked on Trial Friend, including Amyotrophic Lateral Sclerosis, Focal Segmental Glomerulosclerosis, Geographic Atrophy and 8 more, with 15 active clinical trials (11 recruiting) and 1 FDA-approved rare disease drug.
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), given accelerated approval in November 2025 to lower protein in the urine of adults with primary IgA nephropathy at risk of getting worse. The company also has repinatrabit in Phase 3 for phenylketonuria (the trial started in October 2025).
Otsuka Pharmaceutical has 15 active clinical trials across 4 development stages, with 11 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.
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.
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RECRUITINGPHASE1Recently updatedNCT07713758
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
This trial is intended to understand how the body processes repinatrabit in people with moderate liver impairment and severe kidney impairment and also to compare these results with people of similar age, weight, and sex who have normal liver function.
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)
The goal of this Phase 3, randomized study is to assess the safety, efficacy, tolerability, and pharmacokinetics (PK) of oral JNT-517 in adults (18 years of age or older) with PKU. Participants will receive either JNT-517 or placebo and will be blinded to their treatment assignment. Participants will have a 2 in 3 (or approximately 67%) chance of receiving JNT-517 during the first part of the study which will last approximately six weeks. During the second part of the study every participant who continues in the study will receive one of two doses of JNT-517 for an additional 46 weeks. The study requires a screening period of up to 35 days to ensure dietary stabilization and amino acid levels required to meet study eligibility. In total, participation in the study could last for up to 400 days.
Participants will:
Take 75 mg JNT-517 or 150 mg JNT-517, or a placebo BID (2x per day) for approximately 365 days; Visit the clinic or have a mobile health nurse visit your home for checkups and tests; Collect urine sample at home and bring to clinic on specified days; Keep a food diary 3 days before each study visit
The goal of this Phase 3, open-label study is to evaluate the long-term safety of JNT-517 in pediatric and adult participants with Phenylketonuria (PKU) after completion of either Study JNT517-101 (NCT05781399) or JNT517-201 (NCT06637514) as well as participants who have not participated in a prior JNT-517 study. In this trial, all participants will receive JNT-517 using age- and weight-banded dosing as outlined in the protocol, regardless of any dose received in a previous study.
A Long-term Trial of EB-1020 in Pediatric Patients With ADHD
Intervention:EB-1020 (Centanafadine) low dose, EB-1020 (Centanafadine) high dose
Attention-Deficit Hyperactivity Disorder(ADHD)
The purpose of this study is to evaluate the safety of long-term administration of mainly high doses of EB-1020 over 52 weeks in pediatric ADHD patients.
RECRUITINGPHASE2, PHASE3No updates in a whileNCT07086313
A Trial to Evaluate the Efficacy and Safety of EB-1020 in Pediatric Patients With ADHD
Intervention:EB-1020 (Centanafadine) low dose, EB-1020 (Centanafadine) high dose, Placebo
Attention-Deficit Hyperactivity Disorder(ADHD)
The purpose of this study is to evaluate the efficacy and examine the safety of two doses of EB-1020 QD XR capsule administered once daily orally in pediatric ADHD patients.
The purpose of this study is to survey the safety of LUPKYNIS in patients with lupusnephritis under actual use conditions. In addition, information on efficacy will be collected.
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Otsuka Pharmaceutical FDA-Approved Drugs (1)
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.
Otsuka Pharmaceutical clinical trials are running at 400 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.
United States
89▼
Japan
40▼
Australia
27▼
China
25▼
Spain
22▼
South Korea
17▼
France
16▼
India
15▼
Italy
14▼
Brazil
11▼
Canada
10▼
United Kingdom
9▼
Rare Disease Focus Areas (11)
Diseases targeted by Otsuka Pharmaceutical's clinical trial and drug development programs
Pharmaceutical and biotech companies working on similar rare diseases. Overlapping programs can mean more treatment options and competing trials for patients with these conditions.
Frequently Asked Questions About Otsuka Pharmaceutical
Common questions about Otsuka Pharmaceutical's rare disease programs, clinical trials, and treatments.
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), given accelerated approval in November 2025 to lower protein in the urine of adults with primary IgA nephropathy at risk of getting worse. The company also has repinatrabit in Phase 3 for phenylketonuria (the trial started in October 2025).
Otsuka Pharmaceutical has active programs across 11 rare diseases: Amyotrophic Lateral Sclerosis, Focal Segmental Glomerulosclerosis, Geographic Atrophy, IgA Nephropathy, IgA Vasculitis with Nephritis, Membranous Nephropathy, Myelodysplastic Syndromes, Phenylketonuria, Polycystic Kidney Disease, Sjögren's Syndrome, SystemicSclerosis. 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 Amyotrophic Lateral Sclerosis, Focal Segmental Glomerulosclerosis, Geographic Atrophy. 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 has 1 FDA-approved drug with a rare disease use that we track, as of October 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.
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.