Merck & Co., Inc.
Merck & Co., Inc. works on 43 rare diseases tracked on Trial Friend, including Adrenocortical Carcinoma, Aplastic Anemia, Charcot-Marie-Tooth Disease and 40 more, with 20 active clinical trials (4 recruiting) and 8 FDA-approved rare disease drugs.
Merck is a global pharmaceutical company with a substantial rare disease presence through oncology and cardiovascular programs. The company's Keytruda franchise covers rare cancers including Merkel cell carcinoma and mesothelioma, while Winrevair (sotatercept) became the first activin signaling inhibitor approved for pulmonary arterial hypertension in 2024.
Focus areas at Merck & Co., Inc.
Within its broader pharmaceutical portfolio, Merck & Co., Inc. has active clinical trial programs and drug development efforts across 43 rare diseases, including Adrenocortical Carcinoma, Aplastic Anemia, Charcot-Marie-Tooth Disease, Cholangiocarcinoma, Chronic Graft-versus-Host Disease, and 38 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 active and recruiting rare disease studies sponsored by Merck & Co., Inc., 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.
Merck & Co., Inc. is headquartered in Rahway, United States, founded in 1891, publicly traded under the ticker symbol MRK. 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.
Merck & Co., Inc. Drug Pipeline
Merck & Co., Inc. has 20 active clinical trials across 4 development stages, with 4 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.
Merck & Co., Inc. Clinical Trials (20)
Active and recruiting clinical trials sponsored by Merck & Co., Inc., 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.
Merck & Co., Inc. FDA-Approved Drugs (8)
Medications developed or marketed by Merck & Co., Inc. 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 |
|---|---|---|---|
| RALTEGRAVIR | ISENTRESS oral | Oct 12, 2007 | |
| PEMBROLIZUMAB Programmed Death Receptor-1 Blocking Antibody [EPC] | KEYTRUDA intravenous | Sep 4, 2014 | |
| PEMBROLIZUMAB AND BERAHYALURONIDASE ALFA-PMPH Programmed Death Receptor-1 Blocking Antibody [EPC] | KEYTRUDA QLEX subcutaneous | Sep 19, 2025 | |
| POSACONAZOLE Azole Antifungal [EPC] | NOXAFIL oral, intravenous | Sep 15, 2006 | |
| BELZUTIFAN Hypoxia-inducible Factor Inhibitor [EPC] | WELIREG oral | Aug 13, 2021 |
Merck & Co., Inc. Trial Locations
Merck & Co., Inc. clinical trials are running at 706 sites in 38 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 (43)
Diseases targeted by Merck & Co., Inc.'s clinical trial and drug development programs
Adrenocortical carcinoma is a rare, aggressive cancer of the adrenal cortex, the outer layer of the adrenal glands that sit on top of each kidney. These glands produce essential hormones including cor...
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...
Charcot-Marie-Tooth disease (CMT) is the most common inherited disease of the peripheral nervous system. The peripheral nerves that connect the spinal cord to the muscles in the feet, legs, and hands ...
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 (...
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...
Patient Resources
Organizations and resources related to Merck & Co., Inc.'s rare disease focus areas
Frequently Asked Questions About Merck & Co., Inc.
Common questions about Merck & Co., Inc.'s rare disease programs, clinical trials, and treatments.