Janssen / Johnson & Johnson
Janssen / Johnson & Johnson works on 22 rare diseases tracked on Trial Friend, including Aplastic Anemia, Chronic Graft-versus-Host Disease, Cystic Fibrosis and 19 more, with 29 recruiting clinical trials and 25 FDA-approved rare disease drugs.
Janssen, the pharmaceutical division of Johnson & Johnson, operates globally with a focus on various therapeutic areas including treatments for rare neurological and genetic disorders. In April 2025, the FDA approved IMAAVY (nipocalimab), a first-in-class FcRn blocker for generalized myasthenia gravis in patients 12 and older.
Focus areas at Janssen / Johnson & Johnson
Within its broader pharmaceutical portfolio, Janssen / Johnson & Johnson has active clinical trial programs and drug development efforts across 22 rare diseases, including Aplastic Anemia, Chronic Graft-versus-Host Disease, Cystic Fibrosis, Dermatomyositis, Focal Epilepsy, and 17 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 Janssen / Johnson & Johnson, 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.
Janssen / Johnson & Johnson is headquartered in New Brunswick, United States, founded in 1886, publicly traded under the ticker symbol JNJ. 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.
Janssen / Johnson & Johnson Drug Pipeline
Janssen / Johnson & Johnson has 29 active clinical trials across 4 development stages, with 29 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 Janssen / Johnson & Johnson'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.
Janssen / Johnson & Johnson Clinical Trials (29)
Active and recruiting clinical trials sponsored by Janssen / Johnson & Johnson, 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.
Janssen / Johnson & Johnson FDA-Approved Drugs (25)
Medications developed or marketed by Janssen / Johnson & Johnson 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 |
|---|---|---|---|
| DARATUMUMAB AND HYALURONIDASE-FIHJ (HUMAN RECOMBINANT) CD38-directed Cytolytic Antibody [EPC] | Darzalex Faspro subcutaneous | May 1, 2020 | |
| RILPIVIRINE HYDROCHLORIDE | EDURANT oral | Mar 15, 2024 | |
| NIPOCALIMAB-AAHU | IMAAVY intravenous | Apr 29, 2025 | |
| INFLIXIMAB Tumor Necrosis Factor Blocker [EPC] | INFLIXIMAB intravenous | Aug 24, 1998 | |
| GEMCITABINE INTRAVESICAL Nucleoside Metabolic Inhibitor [EPC] | INLEXZO intravesical | Sep 9, 2025 |
Janssen / Johnson & Johnson Trial Locations
Janssen / Johnson & Johnson clinical trials are running at 1,006 sites in 37 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 (22)
Diseases targeted by Janssen / Johnson & Johnson's clinical trial and drug development programs
Aplastic anemia is a rare bone marrow failure syndrome characterized by pancytopenia resulting from absent or severely reduced hematopoiesis. Approximately half are immune-mediated, while others resul...
Chronic graft-versus-host disease is an immune-mediated complication after allogeneic stem cell or bone marrow transplant, in which donor immune cells attack the recipient's tissues. It can affect the...
Cystic fibrosis is an autosomal recessive genetic disorder affecting the CFTR protein, which normally regulates chloride transport. Defective CFTR causes thick, sticky secretions in the lungs and dige...
Dermatomyositis is a rare autoimmune inflammatory disease causing muscle weakness and distinctive skin rashes, particularly over joints. It involves inflammation of muscles and skin blood vessels. Abo...
Focal epilepsy is a neurological disorder characterized by recurrent seizures that originate in a specific area of the brain. It is the most common form of epilepsy in adults. Seizures may involve alt...
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...
Patient Resources
Organizations and resources related to Janssen / Johnson & Johnson's rare disease focus areas
Frequently Asked Questions About Janssen / Johnson & Johnson
Common questions about Janssen / Johnson & Johnson's rare disease programs, clinical trials, and treatments.