Regeneron Pharmaceuticals
Drugmaker with rare disease work · based in Tarrytown, United States · founded 1988 · ticker REGN · regeneron.com
Regeneron Pharmaceuticals works on 20 rare diseases we track, including Acute Myeloid Leukemia, Amyotrophic Lateral Sclerosis and 18 more. It has 23 active clinical trials, and 19 are looking for volunteers now. It also has 5 FDA-approved rare disease drugs.
The FDA is due to decide on Cemdisiran for Generalized myasthenia gravis (anti-AChR antibody-positive adults) in November 2026.
Email me the day the FDA decidesAll upcoming rare disease FDA decisions
Regeneron Pharmaceuticals is a Tarrytown, New York biotech best known for antibody medicines. Its rare disease medicines include Evkeeza (evinacumab) for homozygous familial hypercholesterolemia, approved in February 2021, Veopoz (pozelimab) for CHAPLE disease, approved in August 2023, and Lynozyfic (linvoseltamab) for relapsed or refractory multiple myeloma, approved in July 2025.
Regeneron Pharmaceuticals pipeline
Each row lists the diseases Regeneron Pharmaceuticals is studying at that stage, from the ones closest to approval down to the earliest work.
Evkeeza (evinacumab-dgnb) · Lynozyfic (linvoseltamab-gcpt) · Pasatru (garetosmab-grts) · Praluent (alirocumab) · Veopoz (pozelimab)
Cemdisiran for Generalized myasthenia gravis (anti-AChR antibody-positive adults), with a decision due in November 2026.
Multiple Myeloma 3 trials recruiting · ATTR Amyloidosis (Transthyretin Amyloidosis) recruiting · Eosinophilic Esophagitis recruiting · Paroxysmal Nocturnal Hemoglobinuria recruiting · B-Cell Non-Hodgkin Lymphoma (B-NHL)
Multiple Myeloma 7 trials recruiting · Juvenile Idiopathic Arthritis recruiting · Amyotrophic Lateral Sclerosis recruiting · Recurrent Diffuse Large B-Cell Lymphoma recruiting · Non-Transfusion Dependent Beta-Thalassemia (NTDT)
Multiple Myeloma recruiting · Myelodysplastic Syndromes recruiting · Merkel Cell Carcinoma recruiting
Multiple Myeloma recruiting
Regeneron Pharmaceuticals clinical trials
These are Regeneron Pharmaceuticals 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.
19 trials looking for volunteers now
A Trial to Learn How Well Linvoseltamab Works Compared to the Combination of Elotuzumab, Pomalidomide and Dexamethasone for Adult Participants With Relapsed/Refractory Multiple Myeloma
Tests Linvoseltamab, Elotuzumab, Pomalidomide, Dexamethasone
For Relapsed Refractory Multiple Myeloma (RRMM)
A Trial to Study if REGN17372 in Combination With Linvoseltamab is Tolerable for Adult Participants With Relapsed/Refractory Multiple Myeloma
Tests Linvoseltamab, REGN17372+Linvoseltamab
For Relapsed Refractory Multiple Myeloma (RRMM)
A Study to Examine the Effects of Novel Therapy Linvoseltamab in Combination With Other Cancer Treatments for Adult Participants With Multiple Myeloma That is Resistant to Current Standard of Care Treatments
Tests Linvoseltamab, Daratumumab, Carfilzomib, Lenalidomide, Bortezomib, Pomalidomide, Isatuximab, Fianlimab, Cemiplimab, Nirogacestat, Cevostamab
For Multiple Myeloma
A Study to Compare Linvoseltamab Monotherapy and Linvoseltamab + Carfilzomib Combination Therapy With Standard-of-Care Combination Regimens in Adult Participants With Relapsed/Refractory Multiple Myeloma (RRMM)
Tests Linvoseltamab, Carfilzomib, Daratumumab, Dexamethasone, Pomalidomide, Bortezomib
For Relapsed and/or Refractory Multiple Myeloma (RRMM)
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
Tests NTLA-2001, Placebo
For Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Adult Outpatient Linvoseltamab With Tocilizumab Prophylaxis to Mitigate the Risk of Cytokine Release Syndrome (CRS)
Tests Linvoseltamab, Tocilizumab
For Relapsed/Refractory Multiple Myeloma (RRMM)
Safety and Tolerability of REGN17235 in Adult Participants With Clonal Cytopenia of Undetermined Significance and Low-Risk Myelodysplastic Syndrome With SF3B1 Mutation
Tests REGN17235
For Clonal Cytopenia of Undetermined Significance (CCUS) and Low-Risk Myelodysplastic Syndrome (LR-MDS)
Phase 1/2 Study of Linvoseltamab in Adult Patients With Relapsed or Refractory Multiple Myeloma
Tests Linvoseltamab
For Multiple Myeloma
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- Trial Alerts, when a new trial opens or a trial changes
- Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month
FDA-approved drugs from Regeneron Pharmaceuticals
From the openFDA database. Tap a drug for side effects, dosing and help paying.
Evkeeza evinacumab-dgnb
For Homozygous Familial Hypercholesterolemia. Approved Feb 11, 2021.
Lynozyfic linvoseltamab-gcpt
For Multiple Myeloma. Approved Jul 2, 2025.
Pasatru garetosmab-grts
For Fibrodysplasia Ossificans Progressiva. Approved Aug 19, 2026. Taken by intravenous route.
Praluent alirocumab
For Homozygous Familial Hypercholesterolemia. Approved Jul 24, 2015. Taken by subcutaneous route.
Veopoz pozelimab
Approved Aug 18, 2023. Taken by intravenous, subcutaneous route.
More about Regeneron Pharmaceuticals
In April 2026 the FDA granted accelerated approval to Otarmeni (lunsotogene parvec), a gene therapy for children and adults with severe to profound hearing loss caused by changes in both copies of the OTOF gene, and in August 2026 it approved Pasatru (garetosmab) for adults with fibrodysplasia ossificans progressiva. With Sanofi, Regeneron also developed Dupixent, which is approved for eosinophilic esophagitis and bullous pemphigoid.
Where Regeneron Pharmaceuticals trials take place
The trials above run at 812 study sites in 42 countries. Tap a country to see its states, cities and hospitals.
Rare diseases Regeneron Pharmaceuticals works on
Each disease page lists every open trial, not only Regeneron Pharmaceuticals's, plus approved treatments.
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
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord.
How common: About 5,000 new cases per year in the U.S.; CDC's National ALS Registry estimates about 33,000 Americans were living with ALS in 2022, a number projected to pass 36,000 by 2030
ATTR Amyloidosis (Transthyretin Amyloidosis)
ATTR amyloidosis is a rare progressive disease where abnormal transthyretin protein accumulates as amyloid deposits in the heart, nerves, and other organs.
How common: Approximately 5,000 to 7,000 new cases diagnosed annually in the U.S
Bullous pemphigoid is an autoimmune blistering disorder caused by autoantibodies against basement membrane proteins, particularly BP180 and BP230.
How common: Approximately 2-23 per million people per year; incidence increases sharply with age
Eosinophilic Esophagitis is a rare allergic/immune-mediated inflammatory disease of the esophagus characterized by eosinophil infiltration causing progressive narrowing and dysfunction.
How common: About 142.5 per 100,000 people in the United States, about 1 in 700 or an estimated 472,380 people, in a 2025 insurance claims study (Thel et al., Clin Gastroenterol Hepatol 2025)
Fibrodysplasia Ossificans Progressiva
Fibrodysplasia Ossificans Progressiva (FOP) is an extremely rare genetic disorder characterized by progressive heterotopic ossification, where soft tissues (muscles, tendons, ligaments) gradually...
How common: Extremely rare, about 1 in 1 million to 1 in 2 million people worldwide
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
Graves' disease is the most common cause of hyperthyroidism, an autoimmune condition where antibodies stimulate the thyroid gland to produce excessive thyroid hormone.
How common: Affects about 1 in 200 people; most common in women ages 30-50
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Regeneron Pharmaceuticals's own site is regeneron.com, and our help paying for treatment page lists drugmaker programs and charity funds.
- NIH GARD - Bullous PemphigoidBullous Pemphigoid and Hemophilia B and 2 more
- National Cancer Institute - Merkel Cell CarcinomaMerkel Cell Carcinoma and Mesothelioma
- NCI SEER Cancer Stat Facts: Acute Myeloid LeukemiaAcute Myeloid Leukemia
- NCI PDQ: Acute Myeloid Leukemia Treatment, patient versionAcute Myeloid Leukemia
- The ALS AssociationAmyotrophic Lateral Sclerosis
- ALS Therapy Development InstituteAmyotrophic Lateral Sclerosis
- StatPearls - Transthyretin Amyloid CardiomyopathyATTR Amyloidosis (Transthyretin Amyloidosis)
- Amyloidosis Research ConsortiumATTR Amyloidosis (Transthyretin Amyloidosis)