Zero ad trackers.We never sell your data.Healthcare professional?Clinician Resources

Novo Nordisk

Drugmaker with rare disease work · based in Bagsværd, Denmark · founded 1989 · ticker NVO · novonordisk.com

Novo Nordisk works on 22 rare diseases we track, including Achondroplasia, Acute Myeloid Leukemia and 20 more. It has 18 active clinical trials, and 11 are looking for volunteers now. It also has 8 FDA-approved rare disease drugs.

Novo Nordisk, formed in 1989 from the merger of Nordisk Insulinlaboratorium (founded 1923) and Novo Terapeutisk Laboratorium (founded 1925), is best known for diabetes and obesity medicines and also runs a rare blood and growth disorder business. Its hemophilia medicines include Alhemo (concizumab), a daily injection under the skin approved for people 12 and older with hemophilia A or B with or without inhibitors, and Rebinyn, a long-acting factor IX.

Novo Nordisk pipeline

Each row lists the diseases Novo Nordisk is studying at that stage, from the ones closest to approval down to the earliest work.

Approved
Drugs the FDA has approved.

Alhemo (concizumab) · Norditropin (somatropin) · Rivfloza (nedosiran) · Sogroya (somapacitan-beco) · Esperoct (antihemophilic factor (recombinant), glycopegylated-exei) · Novoeight (antihemophilic factor recombinant) · NovoSeven RT (coagulation factor viia (recombinant)) · Rebinyn (coagulation factor ix recombinant, glycopegylated)

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

Sickle Cell Disease 3 trials recruiting · Hemophilia A 5 trials recruiting · Wild-Type ATTR Cardiac Amyloidosis recruiting · Turner Syndrome · Noonan Syndrome

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

Sickle Cell Disease 2 trials recruiting

Phase 1
First studies in people, mostly checking safety.
Other studies
Studies with no phase listed, such as registries and natural history studies.

Hematologic Disorder recruiting · Type 2 Diabetes recruiting · ATTR Amyloidosis (Transthyretin Amyloidosis) recruiting

Novo Nordisk clinical trials

These are Novo Nordisk 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.

11 trials looking for volunteers now

Looking for volunteersPhase 3Recently updatedNCT07830732

A Research Study Investigating Denecimig (Mim8) in Adults With Acquired Haemophilia A

Tests Denecimig

For Haemophilia A

The purpose of this study is to find out if denecimig is safe and effective for treating people who have acquired haemophilia A. Participants will be in this study for up to around 1 year and 5 months (73 week).

Ages 18 Years+83 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT07207811

CLEOPATTRA: A Research Study to Look at the Effects of Treatment With a Medicine Called Coramitug (NNC6019-0001) in People With Heart Failure Due to Transthyretin Amyloid (ATTR) Amyloidosis

Tests NNC6019-0001, Placebo (NNC6019-0001)

For Transthyretin Amyloid Cardiomyopathy (ATTR CM)

This study will find out if a new medicine called NNC6019-0001 can help reduce the risk of heart-related death and illness in participants with a condition called transthyretin amyloid cardiomyopathy (ATTR-CM), which affects the heart. Participants will either receive NNC6019-0001 or a placebo (a treatment with no active medicine), and which one they get is decided by chance. Everyone in the study will continue receiving their usual heart treatments as recommended by their doctor.

Ages 18 Years+299 locations
View full study
Looking for volunteersPhase 1Recently updatedNCT07220564

A Research Study Looking at How Different Doses of Study Medicine (Inno8) Work in the Body of People With Haemophilia A

Tests NNC0442-0344 A

For Haemophilia A

This study will test how different doses of study medicine (Inno8) work in the bodies of people with haemophilia A. The purpose of the study is to see if Inno8 is safe to use for people with haemophilia A. The study medicine is a new medicine that cannot yet be prescribed by doctors. The study will last for about 11 weeks.

Ages 18 Years - 64 Years33 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT06609226

A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

Tests Etavopivat A, Etavopivat B, Etavopivat C

For Sickle Cell Disease and Thalassemia

Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant's country.

Ages 2 Years+107 locations
View full study
Looking for volunteersPhase 2Recently updatedNCT06198712

A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease

Tests Etavopivat

For Sickle Cell Disease

The purpose of this study is to evaluate the pharmacokinetics and safety of etavopivat in paediatric participants with sickle cell disease (SCD). Participants will receive etavopivat and will be enrolled in a staggered manner, starting with the oldest age group and followed sequentially by younger cohorts after review of pharmacokinetic and safety data from the preceding cohort. All participants will undergo a 24-week primary treatment period followed by a 72-week extension treatment period to further evaluate long-term safety and pharmacokinetics of etavopivat. The total duration of the study will be approximately 96 weeks.

Ages 6 Months - 18 Years18 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT06612268

A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease

Tests Etavopivat, Placebo

For Sickle Cell Disease

This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo. Which treatment the participants will get is decided by chance. Etavopivat is a new medicine and is currently being tested in other studies in addition to this one. The study will last for about 2 years.

Ages 12 Years+175 locations
View full study
Looking for volunteersPhase 2Recently updatedNCT05953584

A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke

Tests Etavopivat, Hydroxyurea

For Sickle Cell Disease

The study will test a new medicine, etavopivat, for sickle cell disease and see if it is safe and help-ful for participants with sickle cell disease who are at an increased risk of stroke. Participants will be divided into two cohorts depending on their transcranial doppler (TCD) ultrasound results and whether or not they receive hydroxyurea (medication that they may already be taking). In one cohort, participants with conditional transcranial doppler (TCD) or participants with abnormal TCD who are not able to receive hydroxyurea will be included. The study doctor will determine if the TCD result is conditional or abnormal. In another cohort, participants with conditional TCD or participants with abnormal TCD who are receiving a stable dose of hydroxyurea will be included. The study doctor will determine if the TCD result is conditional or abnormal. The participant will start a 52-week (1 year) treatment period. The participant will take 400 milligrams (mg) of etavopivat once a day for the 52 weeks. The dose of 400 mg will be taken as 2 tablets by mouth, each containing 200 mg of etavopivat. Etavopivat may be taken with or without food. Each dose should be taken with a glass of water. As part of the study, the participants will be asked to visit the clinic frequently. The participant will have the opportunity to participate in a 48-week optional extension treatment period. The optional extension treatment period will allow continued as-sessment of safety of etavopivat in paediatric patients. At the end of the study, if deemed appro-priate the participant, the caregiver, and the study doctor, the participant may be offered the op-portunity to participate in a separate study to continue receiving etavopivat. If/when this separate study becomes available, the participant may only transfer to the new study after completion of the 52-week primary treatment period and at any time during the 48-week optional extension treatment period.

Ages 12 Years - 16 Years9 locations
View full study
Looking for volunteersRecently updatedNCT04398628

ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

For Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia and Thrombosis

In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have not yet demonstrated long-term safety and effectiveness beyond the pivotal trials that led to their approval. In addition, results from well controlled, pivotal studies often cannot be replicated once a therapy has been approved for general use.2,3,4,5 In 2019 alone, the FDA has issued approvals for 24 new therapies for congenital and acquired hematologic conditions.6 In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.7 With this increase in potential new therapies possible, it is imperative that clinicians and clinical researchers in the field of non-neoplastic hematology have a uniform, secure, unbiased, and enduring method to collect long-term safety and efficacy data. As emphasized in a recently published review, accurate, uniform and quality national data collection is critical in clinical research, particularly for longitudinal cohort studies covering a lifetime of biologic risk.8

Ages not specified76 locations
View full study
Ask about Novo Nordisk's trials
Answers with sources, in plain English
Start withor ask
Follow a condition Novo Nordisk is studying

Pick a condition. One email when a new trial opens, a trial changes or the FDA decides, from any company, not only Novo Nordisk. Unsubscribe anytime.

You'll get, for Achondroplasia:
  • 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
Turn either off anytime from the link in every email.
We never share your email. Unsubscribe anytime. Privacy policy · Check any health site

FDA-approved drugs from Novo Nordisk

From the openFDA database. Tap a drug for side effects, dosing and help paying.

Alhemo concizumab

For Hemophilia A and Hemophilia B. Approved Dec 20, 2024. Taken by subcutaneous route.

Norditropin somatropin

For Prader-Willi Syndrome, Noonan Syndrome and Turner Syndrome. Approved Jun 20, 2000. Taken by subcutaneous route.

Rivfloza nedosiran

For Primary Hyperoxaluria. Approved Sep 29, 2023. Taken by subcutaneous route.

Sogroya somapacitan-beco

For Noonan Syndrome. Approved Aug 28, 2020. Taken by subcutaneous route.

Esperoct antihemophilic factor (recombinant), glycopegylated-exei

For Hemophilia A.

Novoeight antihemophilic factor recombinant

For Hemophilia A.

NovoSeven RT coagulation factor viia (recombinant)

For Hemophilia A.

Rebinyn coagulation factor ix recombinant, glycopegylated

For Hemophilia B.

More about Novo Nordisk

Its growth hormones Norditropin and Sogroya are approved for growth hormone deficiency and for children with conditions such as Noonan syndrome, and Rivfloza (nedosiran), which came with its 2021 purchase of Dicerna, treats primary hyperoxaluria type 1.

In development are denecimig (Mim8) for hemophilia A, whose FDA review Novo said on October 2, 2026 had been extended with no new date, etavopivat for sickle cell disease and thalassemia, and coramitug for ATTR amyloid cardiomyopathy, both in Phase 3.

Where Novo Nordisk trials take place

The trials above run at 1,607 study sites in 58 countries. Tap a country to see its states, cities and hospitals.

United States
408▼
Italy
84▼
Japan
84▼
France
78▼
Spain
76▼
India
71▼
United Kingdom
68▼
Turkey (Türkiye)
61▼
China
60▼
Poland
50▼
Canada
45▼
Germany
38▼

Rare diseases Novo Nordisk works on

Each disease page lists every open trial, not only Novo Nordisk's, plus approved treatments.

Achondroplasia

Achondroplasia is the most common form of short-limbed dwarfism, caused by a mutation in the FGFR3 gene that disrupts the conversion of cartilage to bone in the growth plates.

How common: Approximately 1 in 25,000 live births; estimated 25,000 to 30,000 people in the United States

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

Alpha-1 Antitrypsin Deficiency

Alpha-1 antitrypsin deficiency is a genetic disorder affecting the lungs and liver, caused by insufficient production of the protective protein alpha-1 antitrypsin.

How common: 1 in 1,500 to 3,500 people of European ancestry; about 100,000 people in the U.S

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

Cholangiocarcinoma

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.

How common: About 8,000 new cases per year in the U.S.; rising incidence worldwide

Chronic Graft-versus-Host Disease

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.

How common: Affects 30-70% of patients who receive allogeneic hematopoietic stem cell transplant; about 14,000 Americans were living with chronic GVHD in 2016, according to a claims study funded in part by Kadmon (Bachier et al., Transplantation and Cellular Therapy, 2021)

Cystic Fibrosis

Cystic fibrosis is an autosomal recessive genetic disorder affecting the CFTR protein, which normally regulates chloride transport.

How common: Nearly 40,000 children and adults in the U.S.; 1 in 2,500 to 3,500 births among Caucasians

Hemophilia A

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

How common: 1 in 4,000 to 5,000 males worldwide; very rare in females

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

Medullary Thyroid Cancer

Medullary thyroid cancer (MTC) is a rare neuroendocrine tumor arising from the parafollicular C cells of the thyroid gland, accounting for 2-4% of all thyroid cancers.

How common: Approximately 1,000-1,200 new cases per year in the U.S.; 2-4% of all thyroid cancers

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)

Neuroendocrine Tumors

Neuroendocrine tumors (NETs) grow from hormone-making cells, most often in the small intestine, pancreas, rectum or lungs.

How common: About 244,000 people in the US were living with a neuroendocrine neoplasm, the wider group that includes NETs, in 2021, with about 8.5 new diagnoses per 100,000 people a year (Dasari et al., JAMA Network Open 2025); an earlier estimate for NETs alone was about 171,000 in 2014 (JAMA Oncology 2017)

Noonan Syndrome

Noonan syndrome is a common autosomal dominant disorder caused by RAS/MAPK pathway mutations, characterized by distinctive facial features, short stature, cardiac abnormalities, and developmental...

How common: 1 in 1,000 to 2,500 people; one of the most common genetic syndromes

Paraganglioma

Paragangliomas are rare neuroendocrine tumors arising from chromaffin tissue outside the adrenal medulla.

How common: 1 in 300,000 to 1,000,000; ~30% are hereditary

Prader-Willi Syndrome

Prader-Willi Syndrome is a rare genetic disorder affecting chromosome 15 that results in weak muscle tone, low hormone levels, incomplete sexual development, intellectual disability, and a constant...

How common: Approximately 1 in 10,000 to 30,000 live births

Primary Hyperoxaluria

Primary hyperoxaluria is a rare inherited metabolic disorder in which the liver overproduces oxalate, a substance that combines with calcium to form kidney stones and can eventually cause kidney...

How common: 1 to 3 per million people in Europe and North America

Sickle Cell Disease

Sickle cell disease is an inherited blood disorder where hemoglobin polymerizes under low oxygen, causing red blood cells to become rigid, sickle-shaped, and prone to hemolysis and vaso-occlusion.

How common: About 100,000 Americans with SCD; about 1 in 365 Black or African American births and 1 in 16,300 Hispanic American births

Spinal Muscular Atrophy

Spinal muscular atrophy is an autosomal recessive genetic disorder affecting motor neurons in the spinal cord, causing progressive muscle weakness and atrophy.

How common: About 1 in 10,000 births; about 1 in 50 people of European or Asian ancestry are carriers of 1 copy of the gene change

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

Turner Syndrome

Turner syndrome results from complete or partial absence of an X chromosome in females, causing short stature, gonadal dysgenesis, cardiac and renal abnormalities.

How common: 1 in 2,000 to 3,000 live female births

Wild-Type ATTR Cardiac Amyloidosis

Wild-type ATTR cardiac amyloidosis is an age-related disease where normal transthyretin protein spontaneously misfolds and accumulates in the heart, causing progressive heart failure.

How common: Estimated to affect up to 13% of males aged 85 and older; substantially underdiagnosed

X-Linked Hypophosphatemia

X-Linked Hypophosphatemia is a rare genetic bone disorder where the body cannot properly regulate phosphate levels, leading to rickets, bone pain, and skeletal deformities.

How common: About 1 in 20,000 to 25,000 newborns, according to MedlinePlus Genetics

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Novo Nordisk's own site is novonordisk.com, and our help paying for treatment page lists drugmaker programs and charity funds.

Questions about Novo Nordisk