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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.

Approved
Drugs the FDA has approved.

Evkeeza (evinacumab-dgnb) · Lynozyfic (linvoseltamab-gcpt) · Pasatru (garetosmab-grts) · Praluent (alirocumab) · Veopoz (pozelimab)

At the FDA
Waiting on an FDA decision.

Cemdisiran for Generalized myasthenia gravis (anti-AChR antibody-positive adults), with a decision due in November 2026.

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

Multiple Myeloma 3 trials recruiting · ATTR Amyloidosis (Transthyretin Amyloidosis) recruiting · Eosinophilic Esophagitis recruiting · Paroxysmal Nocturnal Hemoglobinuria recruiting · B-Cell Non-Hodgkin Lymphoma (B-NHL)

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

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)

Phase 1
First studies in people, mostly checking safety.
After approval
Studies that follow people already taking an approved drug.

Multiple Myeloma recruiting

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

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

Looking for volunteersPhase 3Recently updatedNCT05730036

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)

This study is researching an experimental drug called linvoseltamab, also called REGN5458. Linvoseltamab has previously been studied by itself (without other cancer drugs) in participants who had advanced multiple myeloma that returned and needed to be treated again after many other therapies had failed. These participants were no longer benefiting from standard medications and had no good treatment options. In that study, some participants who were treated with linvoseltamab had improvement of their myeloma (shrinkage of their tumors), including some participants who had complete responses (that is, the treatment got rid of all evidence of myeloma in their bodies). This study is focused on participants who have multiple myeloma that has returned or needs to be treated again after one to four prior treatments and have standard cancer treatment options available to them. The aim of this study is to see how safe and effective linvoseltamab is compared to a combination of three cancer drugs: elotuzumab, pomalidomide and dexamethasone, (called EPd) in participants who have returned after having received prior treatment that included lenalidomide, a proteosome inhibitor, and (for participants in some countries) a cluster of differentiation 38 (CD38) antibody. Half of the participants in this study will get linvoseltamab, and the other half will get EPd. This study is looking at several other research questions, including: * How long participants benefit from receiving linvoseltamab compared with EPd * How many participants treated with linvoseltamab or EPd have improvement of their multiple myeloma and by how much * What side effects happen from taking linvoseltamab compared to EPd * How long participants live while receiving treatment or after treatment with linvoseltamab compared to EPd * If there is any improvement in pain after treatment with linvoseltamab compared to EPd

Ages 18 Years+159 locations
View full study
Looking for volunteersPhase 1/2Recently updatedNCT07455851

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)

This study is researching a drug called REGN17372 used with another drug called linvoseltamab (each individually called "study drug" or "study drugs" when combined) in participants with relapsed (when a tumor comes back) or refractory (when a tumor does not respond to treatment) multiple myeloma. This study is the first time REGN17372 will be given to humans. The aim of the study is to understand if REGN17372 can be given safely with linvoseltamab, and if so, what dosing regimen should be used for this treatment combination, in comparison with linvoseltamab alone. The study is looking at: * What side effects may happen from taking REGN17372 with linvoseltamab * How well REGN17372 and linvoseltamab, or linvoseltamab alone, work in treating multiple myeloma * What is the best dose of REGN17372 when given with linvoseltamab * How much study drug(s) are in the blood at different times * Whether the body makes antibodies against the study drugs (which could make the study drugs less effective or could lead to side effects) * If and how REGN17372 and linvoseltamab affect the overall quality of life, daily activities, symptoms and treatment side effects based on participant own feedback (Phase 2)

Ages 18 Years+9 locations
View full study
Looking for volunteersPhase 1Recently updatedNCT05137054

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

This study is researching an experimental drug called linvoseltamab in combination with other drugs for the treatment of a blood cancer called multiple myeloma. Linvoseltamab has previously been studied as a single agent (without other cancer treatments) in participants with multiple myeloma that returned after prior therapies and needed to be treated again. In the initial study, some participants treated with linvoseltamab had improvement of their myeloma, including complete responses (no evidence of myeloma in their bodies). This study is the first time linvoseltamab will be combined with other cancer therapies. The main goal is to understand if linvoseltamab can be given safely with other cancer treatments, and if so, what dose of linvoseltamab should be used for each combination. The study is looking at several other research questions, including: * How many participants treated with linvoseltamab in combination with each of the other cancer treatments have improvement of their multiple myeloma * What side effects may happen from taking linvoseltamab together with another cancer treatment * How much study drug is in the blood at different times * Whether the body makes antibodies against the study drug(s) (which could make the study drug(s) less effective or could lead to side effects)

Ages 18 Years+42 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT07222761

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)

This study is researching a drug called linvoseltamab (also called "study drug") either given alone or in combination with another anti-myeloma drug called carfilzomib, compared to several standard treatments for progressive Multiple Myeloma (MM) after at least 1 but no more than 3 prior therapies. The aim of this study is to see if the safety and efficacy of linvoseltamab alone or in combination with carfilzomib can deliver better outcomes (deeper and longer responses that help extend life) than standard treatment options. The study is looking at several other research questions, including: * What side effects may happen from taking the study drug * How much study drug is in the blood at different times * Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)

Ages 18 Years+53 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT06128629

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

To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.

Ages 18 Years - 90 Years138 locations
View full study
Looking for volunteersPhase 4Recently updatedNCT07609940

Adult Outpatient Linvoseltamab With Tocilizumab Prophylaxis to Mitigate the Risk of Cytokine Release Syndrome (CRS)

Tests Linvoseltamab, Tocilizumab

For Relapsed/Refractory Multiple Myeloma (RRMM)

This study is researching whether the use of tocilizumab before the first dose of linvoseltamab will decrease the risk of Cytokine Release Syndrome (CRS) in participants who have Relapsed or Refractory Multiple Myeloma (RRMM) who have already been treated with at least four lines of treatment for their multiple myeloma, including medicines called a proteasome inhibitor, an immunomodulatory drug, and an anti-Cluster of Differentiation (CD) 38 antibody. The aim of the study is to see how safe, tolerable and effective linvoseltamab is when given after tocilizumab. The study is looking at several other research questions, including: * What side effects may happen from taking tocilizumab before the first dose of linvoseltamab * Whether tocilizumab has an impact on CRS, including whether participants require hospital care and, if so, how many hospital visits occur and how long they last * How frequently other medications (for example, corticosteroids or additional doses of tocilizumab) are used to support participants' care if needed

Ages 18 Years+7 locations
View full study
Looking for volunteersPhase 1Recently updatedNCT07753148

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)

This study will test a study drug called REGN17235 (the "study drug") to see if it can help treat Clonal Cytopenia of Undetermined Significance (CCUS) and Low-Risk Myelodysplastic Syndrome (LR-MDS) with a specific genetic mutation (SF3B1 Mutation). The study is looking at: * What side effects the study drug might cause * How well the study drug works * How much of the study drug is in the blood at different times * If the body makes antibodies (proteins that attach to substances your body does not recognize) against the study drug; this may cause the study drug to not work as well. * What is the best dose of the study drug to treat CCUS and LR-MDS

Ages 18 Years+2 locations
View full study
Looking for volunteersPhase 1/2Recently updatedNCT03761108

Phase 1/2 Study of Linvoseltamab in Adult Patients With Relapsed or Refractory Multiple Myeloma

Tests Linvoseltamab

For Multiple Myeloma

The main purpose of this study is to learn about the safety of linvoseltamab and to find out what is the best dose of linvoseltamab to give to patients with multiple myeloma and to look for any signs that linvoseltamab can effectively treat cancer. The study is looking at several other research questions, including: * Side effects that may be experienced by people receiving linvoseltamab * How linvoseltamab works in the body * How much linvoseltamab is present in the blood * How linvoseltamab may work to treat cancer

Ages 18 Years+40 locations
View full study
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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.

United States
136▼
Spain
93▼
Australia
53▼
Brazil
50▼
Italy
45▼
South Korea
44▼
Japan
37▼
France
36▼
United Kingdom
35▼
Taiwan
29▼
Germany
24▼
Turkey (Türkiye)
22▼

Rare diseases Regeneron Pharmaceuticals works on

Each disease page lists every open trial, not only Regeneron Pharmaceuticals'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

Amyotrophic Lateral Sclerosis

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

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

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

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

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

Hemophilia B

Hemophilia B is an X-linked bleeding disorder caused by deficiency or dysfunction of clotting factor IX.

How common: 1 in 25,000 to 33,000 males; very rare in females

Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia is a severe genetic disorder causing extremely high cholesterol levels from birth, leading to early heart disease and cholesterol deposits in tendons and...

How common: Approximately 1 in 250,000 to 1 in 360,000 people globally

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)

Merkel Cell Carcinoma

Merkel cell carcinoma is a rare, aggressive skin cancer that arises from Merkel cells, specialized touch receptors in the skin.

How common: About 3,000 new cases per year in the U.S.; incidence has tripled over the past 2 decades

Mesothelioma

Mesothelioma is a rare, aggressive cancer that develops in the mesothelium, the thin layer of tissue covering most internal organs.

How common: About 3,000 new cases per year in the U.S

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

Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria is a rare, life-threatening blood disorder where complement-mediated destruction of red blood cells occurs due to acquired mutations in the PIGA gene.

How common: Estimated at 1 to 5 per million people (MedlinePlus Genetics); NORD gives 0.5 to 1.5 per million

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)

Thalassemia

Thalassemia is an inherited blood disorder where reduced or absent production of one type of hemoglobin chain causes anemia, from mild or none in carriers to severe in the major forms, where it can...

How common: About 1 in 100,000 people have symptoms of beta thalassemia, according to NORD, most often people with Mediterranean, Middle Eastern, African or Asian roots

Thyroid Eye Disease

Thyroid eye disease (TED) is a rare autoimmune condition in which the immune system attacks the muscles and fat behind the eyes, causing bulging eyes, double vision, pain, and in severe cases vision...

How common: About 16 in 100,000 women and 3 in 100,000 men diagnosed each year; up to half of people with Graves' disease develop some degree of TED

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.

Questions about Regeneron Pharmaceuticals