Bayer AG

Bayer AG works on 25 rare diseases tracked on Trial Friend, including Alport Syndrome, Aplastic Anemia, ATTR Amyloidosis (Transthyretin Amyloidosis) and 22 more, with 27 recruiting clinical trials and 10 FDA-approved rare disease drugs.

Bayer is a global pharmaceutical and life sciences company with a growing oncology portfolio that includes Vitrakvi (larotrectinib), the first FDA-approved treatment specifically targeting NTRK gene fusions across solid tumor types regardless of where the cancer originated. Bayer also maintains active clinical programs in rare hematologic and cardiovascular conditions.

Type
Diversified Pharma
Ticker
BAYN
Headquarters
Leverkusen, Germany
Founded
1863
Website
bayer.com
27
Active Rare Disease Trials
10
Approved Rare Disease Drugs
25
Rare Diseases in Portfolio
163
Years Active

Focus areas at Bayer AG

Within its broader pharmaceutical portfolio, Bayer AG has active clinical trial programs and drug development efforts across 25 rare diseases, including Alport Syndrome, Aplastic Anemia, ATTR Amyloidosis (Transthyretin Amyloidosis), Cholangiocarcinoma, Chronic Graft-versus-Host Disease, and 20 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 Bayer AG, 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.

Bayer AG is headquartered in Leverkusen, Germany, founded in 1863, publicly traded under the ticker symbol BAYN. 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.

Bayer AG Drug Pipeline

Bayer AG has 27 active clinical trials across 5 development stages, with 27 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 Bayer AG'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.

Understand Bayer AG's pipeline
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1
Early Phase / Phase 11 trial
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women
Recruiting
7
Phase 27 trials
Recruiting
Primary Photoreceptor Disease+4 more
Recruiting
High-risk Localized Prostate Cancer
Recruiting
5
Phase 35 trials
Hypertension, Pulmonary
Recruiting
Pediatric+1 more
Recruiting
Metastatic Hormone-Sensitive Prostate Cancer (mHSPC)
Recruiting
1
Phase 4 / Post-Market1 trial
Transthyretin Amyloid Cardiomyopathy
Recruiting
13
Other13 trials

Bayer AG Clinical Trials (27)

Active and recruiting clinical trials sponsored by Bayer AG, 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.

Ask about Bayer AG's trials
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RECRUITINGPHASE4Recently updatedNCT07298044

A Study to Learn More About the Change in the Blood Levels of Transthyretin When Participants With Transthyretin Amyloidosis With Cardiomyopathy Switch From Tafamidis to Acoramidis

Intervention: Acoramidis

Transthyretin Amyloid Cardiomyopathy

Transthyretin (TTR) is a protein made by the liver that helps transport thyroid hormone and vitamin A in the blood. In some people, this protein breaks down and forms harmful clumps called amyloid. TTR amyloid gets deposited in the heart wall and stops it from pumping blood properly, which may lead to heart failure. The breakage in TTR protein can be age-related (wild-type ATTR-CM), or genetic (variant ATTR-CM). The study drug, acoramidis, works by attaching itself to the TTR protein, making TTR more stable and less likely to break down and form amyloid (clumps). This helps to slow down the progression of the disease, improve heart function, and increase the TTR levels in the blood. Acoramidis is an approved treatment for wild-type or variant ATTR-CM in Europe and the United States. Tafamidis is another drug that stabilizes TTR and prevents it from breaking down. In this study, acoramidis will be studied in participants with ATTR--CM who were previously treated with tafamidis. The main purpose of this study is to assess the change in blood TTR levels after participants are switched from tafamidis to acoramidis. This will be studied to understand if acoramidis causes an increase in blood TTR levels beyond the levels achieved with tafamidis. For this, the researchers will measure the change in the levels of TTR protein in participants' blood after 6 months of the treatment with acoramidis, or earlier if a participant stops the treatment before reaching that six-month mark. All participants will continue taking tafamidis during the screening period. In the treatment period of the study, participants will take acoramidis as two tablets twice daily by mouth, for up to 6 months. At the start of this study, the study doctors will review each participant's medical history and check their overall health. The study doctors will perform electrocardiograms (ECG), and measure blood pressure and heart rate. Researchers will also take blood and urine samples from the participants to measure levels of TTr, NT-proBNP, hs-TnT, hs-CRP, RBP4, eGFR, creatinine, cystatin-C, UACR, and TSH at the start of the study, and at various time points thereafter (during the study) to assess heart, kidney and thyroid function. There will be a total of 9 study check-ins. Participants will visit the study site twice: at screening and at the end of treatment period. A study nurse will visit the participant's home 6 times, at the start of treatment, Weeks 1, 2, 3 and 4, then again at 3 months. The final check-in will be done by phone. The study doctors will monitor the health of the participants regularly for any medical problems during follow-up visits. Participants will know the treatment they will receive during the study. Each participant could be in the study for about 8 months.

Ages 18 Years - 90 Years26 locations
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ACTIVE NOT RECRUITINGPHASE3Recently updatedNCT02562235

Riociguat in Children With Pulmonary Arterial Hypertension (PAH)

Intervention: Riociguat (Adempas, BAY63-2521)

Hypertension, Pulmonary

This study was designed to evaluate the safety, tolerability, pharmacodynamics and pharmacokinetics of riociguat at age-, sex- and body-weight-adjusted doses of 0.5 mg, 1.0 mg, 1.5 mg, 2.0 mg and 2.5 mg TID in children from ≥6 to less than 18 years with pulmonary arterial hypertension (PAH) group 1. The study design consisted of a main study part followed by an optional long-term extension part. The main treatment period consisted of two phases: titration phase up to 8 weeks and a maintenance phase up to 16 weeks.

Ages 6 Years - 17 Years16 locations
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ACTIVE NOT RECRUITINGRecently updatedNCT04899661

A Study to Learn More About the Long-term Safety and Effectiveness of Molidustat as a Treatment for Japanese Men and Women With Renal Anemia

Intervention: Molidustat (Musredo, BAY85-3934)

Renal Anemia

In this study, the researchers want to learn more about the safety and effectiveness of Molidustat in Japanese men and women who have renal anemia, a condition in which the kidneys do not make enough of a hormone that helps the body make new red blood cells. In previous clinical trials with a small number of participants, several important adverse events were observed. The researchers in this study want to know how many patients also have these adverse events under the real world, and if the number of the red blood cells will be increased after the treatment of Molidustat. The researchers will collect the participants' health information from their medical records and their regular check-ups for up to 2 years. The data from this study will be submitted to the health authority in Japan in accordance with the local regulation.

Ages 20 Years+1 location
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ACTIVE NOT RECRUITINGPHASE3Recently updatedNCT06081894

A Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM)

Intervention: Aficamten, Placebo

This clinical trial will study the effects of aficamten (versus placebo) on the quality of life, exercise capacity, and clinical outcomes of patients with non-obstructive hypertrophic cardiomyopathy.

Ages 18 Years - 85 Years180 locations
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ACTIVE NOT RECRUITINGPHASE2Recently updatedNCT07211685

A Study to Learn About How Well BAY 3401016 Works in Adults With Alport Syndrome

Intervention: BAY 3401016, Placebo

Alport syndrome (AS) is a rare genetic condition that causes kidney disease, hearing loss, and eye abnormalities that occur due to changes in specific genes (COL4A3, COL4A4, and COL4A5). These genes help in producing an important protein called collagen. People with AS have a high risk of developing chronic kidney disease (CKD), a condition in which there is progressive loss in kidney function over time. The kidneys soon lose their ability to remove waste products from the body properly, resulting in end-stage kidney disease. A common sign of decreasing kidney function is the presence of excess protein in the urine that is not usually found with healthy kidneys. This condition is known as proteinuria. The study drug, BAY 3401016 (a monoclonal antibody), is a type of medicine that blocks a protein called Semaphorin 3A (Sema3A), which is thought to be involved in causing kidney damage in AS. By blocking the action of the Sema3A protein, BAY 3401016 may prevent proteinuria and slow down the loss in kidney function due to AS. The main purpose of this study is to learn more about how well BAY 3401016 works in slowing down the loss in kidney function in adults with a rapidly progressing AS.

Ages 18 Years - 45 Years58 locations
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RECRUITINGRecently updatedNCT07791160

Study of Acoramidis in Patients With Transthyretin Amyloid Cardiomyopathy

Intervention: Acoramidis

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive heart disease caused by the buildup of an abnormal protein, called transthyretin (TTR), in the heart. This buildup can make it harder for the heart to pump blood and may lead to worsening symptoms over time. Acoramidis is a medicine approved for the treatment of adults with wild-type or hereditary (variant) ATTR-CM. While its benefits have been demonstrated in clinical trials, more information is needed about how it is used and how patients do in everyday medical practice. The MOSAIC-TTR study is an observational study in France. Participants will receive acoramidis as part of their usual medical care. No experimental treatments or additional medical procedures will be required. The study will collect information directly from participating hospitals and from the Healthcare European Amyloidosis Registry (HEAR; NCT05101304). The main goal of the study is to understand how patients' quality of life, daily functioning, and overall well-being change during the first 12 months of treatment with acoramidis, using questionnaires completed by the patients themselves. The study will also collect information about the characteristics of patients receiving acoramidis, how the medicine is used in routine clinical practice, and its safety and tolerability. The information collected will help improve the understanding of ATTR-CM and may help improve the care of people living with this condition.

Ages 18 Years+1 location
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RECRUITINGPHASE1, PHASE2Recently updatedNCT06789445

A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)

Intervention: OpCT-001, OpCT-001

Primary Photoreceptor DiseaseRetinitis Pigmentosa (RP)Usher SyndromeInherited Retinal Disease (IRD)Rod-Cone Dystrophy

Study OpCT-001-101 is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 focuses on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups.

Ages 18 Years+5 locations
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Bayer AG FDA-Approved Drugs (10)

Medications developed or marketed by Bayer AG 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 NameBrand NameRare DiseasesApproval Date
PRAZIQUANTEL
Anthelmintic [EPC]
Biltricide
oral
Dec 29, 1982
GADOBUTROL
Gadolinium-based Contrast Agent [EPC]
Gadavist
intravenous
Mar 14, 2011
ESTRADIOL VALERATE AND ESTRADIOL VALERATE/DIENOGESTNataziaMay 6, 2010
LAROTRECTINIB
Kinase Inhibitor [EPC]
VITRAKVI
oral, oropharyngeal
Nov 26, 2018
DROSPIRENONE AND ETHINYL ESTRADIOLYazMar 16, 2006

Bayer AG Trial Locations

Bayer AG clinical trials are running at 570 sites in 39 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
141▼
China
70▼
Japan
66▼
Germany
65▼
Italy
31▼
Spain
24▼
Canada
17▼
United Kingdom
16▼
France
15▼
Austria
10▼
Brazil
10▼
Portugal
10▼

Rare Disease Focus Areas (25)

Diseases targeted by Bayer AG's clinical trial and drug development programs

Alport SyndromeKidney & Renal

Alport Syndrome is a genetic disorder that causes progressive damage to the kidneys, ears, and eyes due to defects in a type of collagen that provides structure and flexibility to tissues. The conditi...

Prevalence: Approximately 1 in 5,000 to 10,000 people worldwide
Aplastic AnemiaBlood & Immune

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...

Prevalence: 1-2 cases per 1 million people per year
ATTR Amyloidosis (Transthyretin Amyloidosis)Metabolic & Lysosomal

ATTR amyloidosis is a rare progressive disease where abnormal transthyretin protein accumulates as amyloid deposits in the heart, nerves, and other organs. It can be inherited (hereditary ATTR) or dev...

Prevalence: Approximately 5,000 to 7,000 new cases diagnosed annually in the U.S.
CholangiocarcinomaRare Cancers

Cholangiocarcinoma is a rare and aggressive cancer that forms in the bile ducts, the thin tubes that carry digestive fluid (bile) from the liver to the small intestine. It can occur inside the liver (...

Prevalence: About 8,000 new cases per year in the U.S.; rising incidence worldwide
Chronic Graft-versus-Host DiseaseBlood & Immune

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...

Prevalence: Affects 30 to 70% of patients who receive allogeneic hematopoietic stem cell transplant; approximately 14,000 new cases annually in the U.S.
Cystic FibrosisPulmonary & Respiratory

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...

Prevalence: About 30,000 people in the U.S.; 1 in 2,500 to 3,500 births among Caucasians

Patient Resources

Organizations and resources related to Bayer AG's rare disease focus areas

Frequently Asked Questions About Bayer AG

Common questions about Bayer AG's rare disease programs, clinical trials, and treatments.