Regeneron Pharmaceuticals
Regeneron Pharmaceuticals works on 23 rare diseases tracked on Trial Friend, including Amyotrophic Lateral Sclerosis, Aplastic Anemia, ATTR Amyloidosis (Transthyretin Amyloidosis) and 20 more, with 23 active clinical trials (15 recruiting) and 5 FDA-approved rare disease drugs.
Regeneron Pharmaceuticals develops therapeutic candidates to treat rare diseases including rare autoinflammatory conditions. The company applies its monoclonal antibody technology and other platforms to address rare eye diseases, allergic and inflammatory conditions, and other rare genetic disorders.
Focus areas at Regeneron Pharmaceuticals
Within its broader pharmaceutical portfolio, Regeneron Pharmaceuticals has active clinical trial programs and drug development efforts across 23 rare diseases, including Amyotrophic Lateral Sclerosis, Aplastic Anemia, ATTR Amyloidosis (Transthyretin Amyloidosis), Bullous Pemphigoid, Diffuse Intrinsic Pontine Glioma, and 18 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 Regeneron Pharmaceuticals, 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.
Regeneron Pharmaceuticals is headquartered in Tarrytown, United States, founded in 1988, publicly traded under the ticker symbol REGN. 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.
Regeneron Pharmaceuticals Drug Pipeline
Regeneron Pharmaceuticals has 23 active clinical trials across 5 development stages, with 15 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.
Regeneron Pharmaceuticals Clinical Trials (23)
Active and recruiting clinical trials sponsored by Regeneron Pharmaceuticals, 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.
Regeneron Pharmaceuticals FDA-Approved Drugs (5)
Medications developed or marketed by Regeneron Pharmaceuticals 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 |
|---|---|---|---|
| AFLIBERCEPT Vascular Endothelial Growth Factor Inhibitor [EPC] | EYLEA HD intravitreal | Aug 18, 2023 | |
| GARETOSMAB-GRTS | Pasatru intravenous | Aug 19, 2026 | |
| ALIROCUMAB PCSK9 Inhibitor [EPC] | Praluent subcutaneous | Jul 24, 2015 | |
| POZELIMAB Complement Inhibitor [EPC] | VEOPOZ intravenous, subcutaneous | — | Aug 18, 2023 |
| LUNSOTOGENE PARVEC-CWHA | OTARMENI intracochlear | — |
Regeneron Pharmaceuticals Trial Locations
Regeneron Pharmaceuticals clinical trials are running at 813 sites in 48 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 (23)
Diseases targeted by Regeneron Pharmaceuticals's clinical trial and drug development programs
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord. This causes progressive weakness and paralysis of voluntary muscles whil...
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...
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...
Bullous pemphigoid is an autoimmune blistering disorder caused by autoantibodies against basement membrane proteins, particularly BP180 and BP230. The condition leads to formation of large fluid-fille...
Diffuse intrinsic pontine glioma (DIPG) is an aggressive childhood brain tumor that grows in the pons, the part of the brainstem controlling breathing, swallowing, and movement. Because it weaves thro...
Eosinophilic Esophagitis is a rare allergic/immune-mediated inflammatory disease of the esophagus characterized by eosinophil infiltration causing progressive narrowing and dysfunction. It presents wi...
Patient Resources
Organizations and resources related to Regeneron Pharmaceuticals's rare disease focus areas
Frequently Asked Questions About Regeneron Pharmaceuticals
Common questions about Regeneron Pharmaceuticals's rare disease programs, clinical trials, and treatments.