Novo Nordisk
Novo Nordisk works on 19 rare diseases tracked on Trial Friend, including Achondroplasia, Alpha-1 Antitrypsin Deficiency, ATTR Amyloidosis (Transthyretin Amyloidosis) and 16 more, with 14 active clinical trials (9 recruiting) and 5 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. 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.
Novo Nordisk Drug Pipeline
Novo Nordisk has 14 active clinical trials across 4 development stages, with 9 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.
Novo Nordisk Clinical Trials (14)
Active and recruiting clinical trials sponsored by Novo Nordisk, 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.
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Novo Nordisk FDA-Approved Drugs (5)
Medications developed or marketed by Novo Nordisk 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 |
|---|---|---|---|
| CONCIZUMAB Tissue Factor Pathway Inhibitor Antagonist [EPC] | Alhemo subcutaneous | Dec 20, 2024 | |
| SOMATROPIN Recombinant Human Growth Hormone [EPC] | Norditropin subcutaneous | Jun 20, 2000 | |
| NEDOSIRAN | RIVFLOZA subcutaneous | Sep 29, 2023 | |
| SOMAPACITAN-BECO | SOGROYA subcutaneous | Aug 28, 2020 | |
| COAGULATION FACTOR IX RECOMBINANT, GLYCOPEGYLATED | REBINYN | — |
Novo Nordisk Trial Locations
Novo Nordisk clinical trials are running at 1,224 sites in 57 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 (19)
Diseases targeted by Novo Nordisk's clinical trial and drug development programs
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. This results in dispropo...
Alpha-1 antitrypsin deficiency is a genetic disorder affecting the lungs and liver, caused by insufficient production of the protective protein alpha-1 antitrypsin. Without adequate protection, neutro...
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...
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 Novo Nordisk's rare disease focus areas
Frequently Asked Questions About Novo Nordisk
Common questions about Novo Nordisk's rare disease programs, clinical trials, and treatments.