Novo Nordisk
Novo Nordisk works on 18 rare diseases tracked on Trial Friend, including Achondroplasia, Alpha-1 Antitrypsin Deficiency, ATTR Amyloidosis (Transthyretin Amyloidosis) and 15 more, with 19 recruiting clinical trials and 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), invests significantly in rare disease drug development. The company's rare disease portfolio includes hemophilia treatments and growth disorder therapies.
Focus areas at Novo Nordisk
Within its broader pharmaceutical portfolio, Novo Nordisk has active clinical trial programs and drug development efforts across 18 rare diseases, including Achondroplasia, Alpha-1 Antitrypsin Deficiency, ATTR Amyloidosis (Transthyretin Amyloidosis), Cholangiocarcinoma, Chronic Graft-versus-Host Disease, and 13 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 all active and recruiting studies sponsored by Novo Nordisk, 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.
Novo Nordisk is headquartered in Bagsværd, Denmark, founded in 1989, publicly traded under the ticker symbol NVO. 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.
Novo Nordisk Drug Pipeline
Novo Nordisk has 19 active clinical trials across 4 development stages, with 19 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 includes all of Novo Nordisk's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
Novo Nordisk Clinical Trials (19)
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.
Novo Nordisk FDA-Approved Drugs (8)
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 | |
| ORAL SEMAGLUTIDE GLP-1 Receptor Agonist [EPC] | OZEMPIC oral | Sep 20, 2019 | |
| NEDOSIRAN | RIVFLOZA subcutaneous | Sep 29, 2023 | |
| SOMAPACITAN-BECO | SOGROYA subcutaneous | Aug 28, 2020 |
Novo Nordisk Trial Locations
Novo Nordisk clinical trials are running at 1,099 sites in 59 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 (18)
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 enzyme alpha-1 antitrypsin. Without adequate protection, neutrop...
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.