Alexion Pharmaceuticals
Alexion Pharmaceuticals works on 31 rare diseases tracked on Trial Friend, including Acromegaly, Amyotrophic Lateral Sclerosis, ATTR Amyloidosis (Transthyretin Amyloidosis) and 28 more, with 33 active clinical trials (17 recruiting) and 5 FDA-approved rare disease drugs.
Alexion, now AstraZeneca's Rare Disease division following a $39 billion acquisition in 2021, is the pioneer of complement-targeted therapies. Key products include Soliris (eculizumab) and its successor Ultomiris (ravulizumab) for paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome, generalized myasthenia gravis, and neuromyelitis optica spectrum disorder. Voydeya (danicopan), the first oral Factor D inhibitor, was FDA-approved in March 2024 as add-on therapy to ravulizumab or eculizumab for clinically significant extravascular hemolysis in adults with PNH. The portfolio also includes Strensiq (asfotase alfa) for hypophosphatasia and Kanuma (sebelipase alfa) for lysosomal acid lipase deficiency.
Alexion Pharmaceuticals Drug Pipeline
Alexion Pharmaceuticals has 33 active clinical trials across 4 development stages, with 17 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.
Alexion Pharmaceuticals Clinical Trials (33)
Active and recruiting clinical trials sponsored by Alexion Pharmaceuticals, 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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Alexion Pharmaceuticals FDA-Approved Drugs (5)
Medications developed or marketed by Alexion Pharmaceuticals 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 |
|---|---|---|---|
| SELUMETINIB | KOSELUGO | Apr 10, 2020 | |
| ECULIZUMAB Complement Inhibitor [EPC] | SOLIRIS intravenous | Mar 16, 2007 | |
| ASFOTASE ALFA Tissue-nonspecific Alkaline Phosphatase [EPC] | STRENSIQ subcutaneous | — | Oct 23, 2015 |
| RAVULIZUMAB Complement Inhibitor [EPC] | Ultomiris intravenous | Dec 21, 2018 | |
| DANICOPAN Complement Factor D Inhibitor [EPC] | Voydeya oral | Mar 29, 2024 |
Alexion Pharmaceuticals Trial Locations
Alexion Pharmaceuticals clinical trials are running at 1,369 sites in 37 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 (31)
Diseases targeted by Alexion Pharmaceuticals'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...
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...
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...
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...
Bullous pemphigoid is an autoimmune blistering disorder caused by autoantibodies against basement membrane proteins, particularly BP180 and BP230. The condition leads to formation of large fluid-fille...
C3 glomerulopathy is a group of rare kidney diseases caused by dysregulation of the alternative complement pathway, leading to uncontrolled C3 deposition in the glomeruli. The two subtypes, C3 glomeru...
Patient Resources
Organizations and resources related to Alexion Pharmaceuticals's rare disease focus areas
Frequently Asked Questions About Alexion Pharmaceuticals
Common questions about Alexion Pharmaceuticals's rare disease programs, clinical trials, and treatments.