Novartis
Latest, July 17, 2026Fabhalta (iptacopan) received full FDA approval for IgA nephropathy on July 16, 2026 after a 24-month trial showed a 48% slower decline in kidney function; Itvisma, the spinal-fluid form of Zolgensma for SMA, was approved November 24, 2025. Novartis press release, July 17, 2026
Novartis works on 80 rare diseases tracked on Trial Friend, including Acromegaly, Alpha-1 Antitrypsin Deficiency, Alport Syndrome and 77 more, with 37 active clinical trials (24 recruiting) and 20 FDA-approved rare disease drugs.
Novartis has one of the broadest rare disease portfolios in the industry, spanning neuroscience, hematology, immunology, and ophthalmology. Key products include Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy, Fabhalta (iptacopan) for paroxysmal nocturnal hemoglobinuria, and Jakavi (ruxolitinib) for myelofibrosis and polycythemia vera.
Focus areas at Novartis
Within its broader pharmaceutical portfolio, Novartis has active clinical trial programs and drug development efforts across 80 rare diseases, including Acromegaly, Alpha-1 Antitrypsin Deficiency, Alport Syndrome, Amyotrophic Lateral Sclerosis, Aplastic Anemia, and 75 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 active and recruiting rare disease studies sponsored by Novartis, 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.
Novartis is headquartered in Basel, Switzerland, founded in 1996, publicly traded under the ticker symbol NOVN. 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.
Novartis Drug Pipeline
Novartis has 37 active clinical trials across 5 development stages, with 24 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.
Novartis Clinical Trials (37)
Active and recruiting clinical trials sponsored by Novartis, 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.
Novartis FDA-Approved Drugs (20)
Medications developed or marketed by Novartis 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 |
|---|---|---|---|
| CRIZANLIZUMAB Selectin Blocker [EPC] | ADAKVEO intravenous | Nov 15, 2019 | |
| EVEROLIMUS Kinase Inhibitor [EPC] | Afinitor oral | Mar 30, 2009 | |
| DEFERASIROX Iron Chelator [EPC] | Exjade oral | Nov 2, 2005 | |
| IPTACOPAN | FABHALTA oral | Dec 5, 2023 | |
| FINGOLIMOD HCL | Gilenya oral | Sep 21, 2010 |
Novartis Trial Locations
Novartis clinical trials are running at 1,692 sites in 61 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 (80)
Diseases targeted by Novartis's clinical trial and drug development programs
Acromegaly is a rare hormonal disorder caused by excessive growth hormone production, typically from a pituitary adenoma, resulting in abnormal growth of hands, feet, and facial features. It also caus...
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...
Alport Syndrome is a genetic disorder that causes progressive damage to the kidneys, ears, and eyes due to defects in a type of collagen that provides structure and flexibility to tissues. The conditi...
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord. This causes progressive weakness and paralysis of voluntary muscles whil...
Aplastic anemia is a rare bone marrow failure syndrome characterized by pancytopenia resulting from absent or severely reduced hematopoiesis. Approximately half are immune-mediated, while others resul...
Atypical Hemolytic Uremic Syndrome (aHUS) is a rare kidney disease caused by uncontrolled activation of the complement system, a part of the immune system that normally helps fight infection. This act...
Patient Resources
Organizations and resources related to Novartis's rare disease focus areas
Frequently Asked Questions About Novartis
Common questions about Novartis's rare disease programs, clinical trials, and treatments.