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Janssen / Johnson & Johnson

Drugmaker with rare disease work · based in New Brunswick, United States · founded 1886 · ticker JNJ · jnj.com

Janssen / Johnson & Johnson works on 21 rare diseases we track, including Acute Myeloid Leukemia, Chronic Graft-versus-Host Disease and 19 more. It has 75 active clinical trials, and 31 are looking for volunteers now. It also has 11 FDA-approved rare disease drugs.

Janssen, the pharmaceutical division of Johnson & Johnson, works across several therapeutic areas, including rare neurological, blood and immune disorders. In April 2025, the FDA approved Imaavy (nipocalimab), an FcRn blocker, for generalized myasthenia gravis in patients 12 and older who are anti-AChR or anti-MuSK antibody positive.

Janssen / Johnson & Johnson pipeline

Each row lists the diseases Janssen / Johnson & Johnson is studying at that stage, from the ones closest to approval down to the earliest work.

Approved
Drugs the FDA has approved.
Phase 3
Large studies, usually the last step before a company asks the FDA for approval.

Multiple Myeloma 12 trials recruiting · Myasthenia Gravis recruiting · Leukemia, Myeloid, Acute recruiting · Geographic Atrophy recruiting · Mantle Cell Lymphoma

Phase 2
Mid-size studies that check whether a drug works and at what dose.

Multiple Myeloma 26 trials recruiting · Non-hodgkin Lymphoma recruiting · Amyloid Light-chain Amyloidosis recruiting · Sickle Cell Disease recruiting · Lymphoma, Non-Hodgkin · Diffuse Intrinsic Pontine Glioma · Leukemia, Myeloid, Acute · Pilomyxoid Astrocytoma · Light Chain Amyloidosis · Mantle Cell Lymphoma · and 2 more

Phase 1
First studies in people, mostly checking safety.

Multiple Myeloma 10 trials recruiting · Lymphoma, Non-Hodgkin 3 trials · Relapsed or Refractory B Cell Non-Hodgkin Lymphoma · Leukemia, Myeloid, Acute

After approval
Studies that follow people already taking an approved drug.

Multiple Myeloma 2 trials recruiting

Other studies
Studies with no phase listed, such as registries and natural history studies.

Janssen / Johnson & Johnson clinical trials

These are Janssen / Johnson & Johnson trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.

31 trials looking for volunteers now

Looking for volunteersPhase 2Recently updatedNCT05998642

Ibrutinib Combination Therapy in Transplant Ineligible Individuals With Newly Diagnosed Primary CNS Lymphoma

Tests Methotrexate, Rituximab (where available), Ibrutinib

For Non-hodgkin Lymphoma

This study is being done to answer the following question: Can the addition of a new drug to the usual treatment lower the chance of primary central nervous system lymphoma growing or spreading? This study is being done to find out if this approach is better or worse than the usual approach for this type of cancer. The usual approach is defined as the care most people get for Primary Central Nervous System Lymphoma (PCNSL).

Ages 18 Years+8 locations
View full study
Looking for volunteersPhase 2Recently updatedNCT06993675

Engaging T-cells to Eliminate MRD in Newly Diagnosed Myeloma Optimizing Response With Talquetmab and Teclistamab (ROTATE)

Tests Talquetamab, Teclistamab

For Multiple Myeloma

Multiple myeloma is characterized by a pattern of recurrent relapse and remains an incurable malignancy. Participants with minimal residual disease (MRD) after front line therapy with induction and transplant have worse prognosis than those with MRD negative disease. Bispecific T-cell-based immunotherapies have the potential to promote further reduction of malignant plasma cells thus improving rates of MRD negativity and improve patient outcomes. In this study, participants who are MRD positive after front line therapy will receive consolidation with GPRC5D-targeted bispecific talquetamab. We will test MRD negative conversion and if MRD negativity was not achieved, the participant will switch to a different target using the B-cell maturation antigen TCE, teclistamab. Consolidation will be continued for up to 1 year in participants who have achieved MRD negativity. After consolidation therapy on this protocol is complete, participants may continue to be treated with standardof- care (SOC) maintenance therapy.

Ages 18 Years+3 locations
View full study
Looking for volunteersPhase 2/3Recently updatedNCT05265273

A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis

Tests Nipocalimab

For Myasthenia Gravis

The purpose of this study is to determine the effect of nipocalimab on total serum immunoglobulin G (IgG) in pediatric participants 2 to less than (\<) 18 years of age (globally) and 8 to \<18 years of age (for Unites Stated (US) sites only), the safety and tolerability of treatment with nipocalimab in children and adolescents and to evaluate the pharmacokinetics (PK) of nipocalimab in children and adolescents with generalized myasthenia gravis (gMG) who have an insufficient clinical response to ongoing, stable standard-of-care therapy.

Ages 2 Years - 17 Years19 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT06852222

A Study of Bleximenib, Venetoclax and Azacitidine For Treatment of Participants With Newly Diagnosed Acute Myeloid Leukemia (AML)

Tests Bleximenib, Venetoclax (VEN), Azacitidine (AZA), Placebo

For Leukemia, Myeloid, Acute

The purpose of this study is to assess how bleximenib and Venetoclax (VEN)+ Azacitidine (AZA) works as compared to placebo and VEN+AZA alone for the treatment of participants with newly diagnosed Acute Myeloid Leukemia (AML) with a mutation in the NPM1 or KMT2A gene.

Ages 18 Years+270 locations
View full study
Looking for volunteersPhase 2Recently updatedNCT07589634

A Study Evaluating the Prophylactic Use of Tocilizumab to Prevent Cytokine Release Syndrome With Ramantamig Administration in Participants With Relapsed/Refractory Multiple Myeloma

Tests Ramantamig, Tocilizumab, Placebo

For Multiple Myeloma

The purpose of this study is to find out whether giving a single dose of tocilizumab before treatment with ramantamig can help prevent or reduce the severity of cytokine release syndrome (CRS) within 28 days from ramantamig, compared to participants who receive placebo. CRS is an acute inflammatory reaction that can occur during treatment and may be associated with flu-like or other systemic symptoms, such as fever and tiredness.

Ages 18 Years+60 locations
View full study
Looking for volunteersPhase 4Recently updatedNCT05201781

A Long-term Study for Participants Previously Treated With Ciltacabtagene Autoleucel

Tests Cilta-cel

For Multiple Myeloma

The purpose of this study is to collect long-term follow-up data on delayed adverse events after administration of ciltacabtagene autoleucel (cilta-cel), and to characterize and understand the long-term safety profile of cilta-cel.

Ages 18 Years+50 locations
View full study
Looking for volunteersPhase 2Recently updatedNCT07149857

A Study to Evaluate Efficacy and Safety of Ciltacabtagene Autoleucel

Tests Cilta-cel, Cyclophosphamide, Induction therapy, Fludarabine

For Multiple Myeloma

The purpose of this study is to evaluate how well (efficacy) cilta-cel works when given with a fludarabine-free lymphodepletion regimen (a process of reducing the number of lymphocytes, a type of white blood cell in the body, typically through chemotherapy), or an alternative administration of cilta-cel infusion following a cyclophosphamide and fludarabine lymphodepletion regimen.

Ages 18 Years - 80 Years20 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT05552222

A Study of Teclistamab in Combination With Daratumumab and Lenalidomide (Tec-DR) and Talquetamab in Combination With Daratumumab and Lenalidomide (Tal-DR) in Participants With Newly Diagnosed Multiple Myeloma

Tests Teclistamab, Daratumumab, Lenalidomide, Dexamethasone, Talquetamab

For Multiple Myeloma

The purpose of this study is to compare the efficacy of teclistamab in combination with daratumumab and lenalidomide (Tec-DR) and talquetamab in combination with daratumumab and lenalidomide (Tal-DR) versus daratumumab, lenalidomide, dexamethasone (DRd).

Ages 18 Years+270 locations
View full study
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FDA-approved drugs from Janssen / Johnson & Johnson

From the openFDA database. Tap a drug for side effects, dosing and help paying.

Darzalex Faspro daratumumab and hyaluronidase-fihj (human recombinant)

For Light Chain Amyloidosis and Multiple Myeloma. Approved May 1, 2020. Taken by subcutaneous route.

Imaavy nipocalimab-aahu

For Myasthenia Gravis and Warm Autoimmune Hemolytic Anemia. Approved Apr 29, 2025. Taken by intravenous route.

Opsumit macitentan

For Pulmonary Arterial Hypertension. Approved Oct 18, 2013.

Ponvory ponesimod

For Multiple Sclerosis. Approved Mar 18, 2021. Taken by mouth.

Talvey talquetamab-tgvs

For Multiple Myeloma. Approved Aug 9, 2023. Taken by subcutaneous route.

Tecvayli teclistamab-cqyv

For Multiple Myeloma. Approved Oct 25, 2022.

Topamax topiramate

For Lennox-Gastaut Syndrome. Approved Dec 24, 1996. Taken by mouth.

Uptravi selexipag

For Pulmonary Arterial Hypertension. Approved Dec 21, 2015.

Ventavis iloprost

For Pulmonary Arterial Hypertension. Approved Dec 29, 2004.

Zavesca miglustat

For Gaucher Disease. Approved Jul 31, 2003.

Carvykti ciltacabtagene autoleucel

For Multiple Myeloma. Taken by intravenous route.

More about Janssen / Johnson & Johnson

In August 2026, Imaavy became the first FDA-approved treatment for warm autoimmune hemolytic anemia, for patients 12 and older who are taking or have taken corticosteroids. Nipocalimab is also in trials for CIDP, Sjögren's disease and pregnancies at risk of hemolytic disease of the fetus and newborn.

Where Janssen / Johnson & Johnson trials take place

The trials above run at 3,710 study sites in 40 countries. Tap a country to see its states, cities and hospitals.

United States
1547▼
Spain
213▼
China
187▼
France
162▼
Japan
138▼
Italy
134▼
Australia
127▼
Germany
124▼
United Kingdom
99▼
Brazil
85▼
Poland
79▼
South Korea
76▼

Rare diseases Janssen / Johnson & Johnson works on

Each disease page lists every open trial, not only Janssen / Johnson & Johnson's, plus approved treatments.

Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a fast-growing cancer of the bone marrow in which immature white blood cells called blasts crowd out normal blood cells, causing fatigue, infections and bleeding...

How common: The National Cancer Institute's SEER program estimates 22,720 new US cases and 11,500 deaths in 2026, about 1.1% of new cancers, and 83,311 people were living with AML in 2023

Chronic Graft-versus-Host Disease

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.

How common: Affects 30-70% of patients who receive allogeneic hematopoietic stem cell transplant; about 14,000 Americans were living with chronic GVHD in 2016, according to a claims study funded in part by Kadmon (Bachier et al., Transplantation and Cellular Therapy, 2021)

Chronic Inflammatory Demyelinating Polyneuropathy

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) is an autoimmune disorder affecting the peripheral nerves, causing progressive weakness and impaired function in the legs and arms.

How common: About 2.8 per 100,000 people in a 2019 pooled analysis of 9 studies (Broers et al., Neuroepidemiology); NORD estimates 5 to 7 per 100,000, and NIH's GARD estimates about 50,000 people in the U.S

Dermatomyositis

Dermatomyositis is a rare autoimmune inflammatory disease causing muscle weakness and distinctive skin rashes, particularly over joints.

How common: About 9.6 new cases per million people a year, according to NORD; juvenile-onset incidence about 0.4 cases per 100,000 children

Focal Epilepsy

Focal epilepsy is a neurological disorder characterized by recurrent seizures that originate in a specific area of the brain.

How common: Up to about 61% of people with epilepsy have focal seizures; about 2.9 million U.S. adults and 456,000 children have active epilepsy

Gastroparesis

Gastroparesis is a condition in which the stomach empties food too slowly even though nothing is blocking it, causing early fullness, nausea, vomiting, bloating and upper belly pain.

How common: In a Minnesota population study cited by the NIDDK, about 10 men and 40 women per 100,000 had diagnosed gastroparesis

Generalized Pustular Psoriasis

Generalized pustular psoriasis (GPP) is the rarest form of psoriasis and behaves like a different disease.

How common: Roughly 2 to 7 new cases per million people a year worldwide (NORD); published estimates range from 2 to 124 per million, lower in Europe and higher in parts of Asia; about half of people with GPP also have ordinary plaque psoriasis

Geographic Atrophy

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...

How common: About 1 million people in the U.S. are estimated to have geographic atrophy, the advanced dry form of age-related macular degeneration

Lennox-Gastaut Syndrome

Lennox-Gastaut Syndrome is a severe childhood epilepsy characterized by multiple types of seizures that are often resistant to medication, intellectual disability, and characteristic EEG findings...

How common: About 1-4% of childhood epilepsy cases, with about 2 new cases per 100,000 children each year (NORD)

Light Chain Amyloidosis

Light Chain Amyloidosis is a rare, serious blood disorder caused by plasma cells producing misfolded immunoglobulin light chains that accumulate in tissues as amyloid fibrils, progressively damaging...

How common: Approximately 600-1,000 new cases per year in the United States; median age of diagnosis is 63 years

Lupus Nephritis

Lupus nephritis is kidney inflammation caused by systemic lupus erythematosus (SLE), in which the immune system attacks the kidneys' filters and, without treatment, can lead to kidney failure.

How common: About 21 per 100,000 people in a Minnesota population study in 2015 (Lupus Midwest Network, 2022)

Mantle Cell Lymphoma

Mantle cell lymphoma is a rare type of non-Hodgkin lymphoma that starts in B cells, a kind of white blood cell.

How common: About 1 in 200,000 people (Cleveland Clinic)

Multiple Myeloma

Multiple myeloma is a cancer of plasma cells, the white blood cells that make antibodies.

How common: About 36,000 new US diagnoses expected in 2026 and roughly 203,000 people living with it, which puts myeloma right at the edge of the US rare disease threshold; it is 1.7% of all new cancers (NCI SEER)

Myasthenia Gravis

Myasthenia gravis is a chronic autoimmune neuromuscular disorder causing muscle weakness and rapid fatigue.

How common: 1 in 5,000 people

Myelodysplastic Syndromes

Myelodysplastic syndromes are a group of blood cancers in which the bone marrow fails to produce enough healthy blood cells.

How common: 10,000 to 15,000 new cases annually in the U.S.; estimated 60,000 to 170,000 Americans living with MDS

Primary Central Nervous System Lymphoma

Primary central nervous system lymphoma (PCNSL) is a fast-growing lymphoma that starts in the brain, spinal cord, eyes or the membranes around them and usually stays there.

How common: About 5 new cases per million people a year in the US (Ono Pharmaceutical and Deciphera); more than half of patients are 60 or older

Pulmonary Arterial Hypertension

Pulmonary arterial hypertension is a rare disease characterized by abnormally high blood pressure in the pulmonary arteries, causing progressive heart and lung damage.

How common: About 26 to 52 per million people in a Scottish national study, with about 7 new cases per million people each year (Peacock et al., European Respiratory Journal, 2007)

Sickle Cell Disease

Sickle cell disease is an inherited blood disorder where hemoglobin polymerizes under low oxygen, causing red blood cells to become rigid, sickle-shaped, and prone to hemolysis and vaso-occlusion.

How common: About 100,000 Americans with SCD; about 1 in 365 Black or African American births and 1 in 16,300 Hispanic American births

Sjögren's Syndrome

Sjögren's syndrome is a chronic autoimmune disorder in which the immune system attacks the moisture-producing exocrine glands, primarily the salivary and lacrimal (tear) glands.

How common: Estimated 1 to 4 million Americans (including undiagnosed); approximately 1 in 1,000 adults based on physician-diagnosed prevalence

Waldenström Macroglobulinemia

Waldenström Macroglobulinemia is a rare blood cancer in which malignant B-lymphocytes produce excessive amounts of a protein called macroglobulin (immunoglobulin M or IgM).

How common: Approximately 1,500 new cases per year in the United States; median age of diagnosis is 68 years

Warm Autoimmune Hemolytic Anemia

Warm autoimmune hemolytic anemia (wAIHA) is a rare autoimmune disease in which the immune system makes antibodies, usually of the IgG type, that stick to red blood cells at normal body temperature...

How common: About 1 to 3 new cases per 100,000 people each year, according to the FDA and NORD

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Janssen / Johnson & Johnson's own site is jnj.com, and our help paying for treatment page lists drugmaker programs and charity funds.

Questions about Janssen / Johnson & Johnson