AstraZeneca PLC
AstraZeneca PLC works on 42 rare diseases tracked on Trial Friend, including Adrenocortical Carcinoma, ATTR Amyloidosis (Transthyretin Amyloidosis), Atypical Hemolytic Uremic Syndrome and 39 more, with 24 recruiting clinical trials and 22 FDA-approved rare disease drugs.
AstraZeneca entered the rare disease market with its 2021 acquisition of Alexion Pharmaceuticals for $39 billion, significantly expanding its portfolio of rare disease treatments. The company now operates a dedicated rare disease division addressing complement-mediated and other rare genetic disorders.
Focus areas at AstraZeneca PLC
Within its broader pharmaceutical portfolio, AstraZeneca PLC has active clinical trial programs and drug development efforts across 42 rare diseases, including Adrenocortical Carcinoma, ATTR Amyloidosis (Transthyretin Amyloidosis), Atypical Hemolytic Uremic Syndrome, Bullous Pemphigoid, Cholangiocarcinoma, and 37 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 AstraZeneca PLC, 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.
AstraZeneca PLC is headquartered in Cambridge, United Kingdom, founded in 1999, publicly traded under the ticker symbol AZN. 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.
AstraZeneca PLC Drug Pipeline
AstraZeneca PLC has 24 active clinical trials across 4 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 includes all of AstraZeneca PLC'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.
AstraZeneca PLC Clinical Trials (24)
Active and recruiting clinical trials sponsored by AstraZeneca PLC, 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.
AstraZeneca PLC FDA-Approved Drugs (22)
Medications developed or marketed by AstraZeneca PLC 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 |
|---|---|---|---|
| ALBUTEROL SULFATE AND BUDESONIDE Corticosteroid [EPC] | AIRSUPRA respiratory (inhalation) | Jan 10, 2023 | |
| TICAGRELOR P2Y12 Platelet Inhibitor [EPC] | BRILINTA oral | Jul 20, 2011 | |
| BUDESONIDE AND FORMOTEROL FUMARATE DIHYDRATE Corticosteroid [EPC] | Budesonide and Formoterol Fumarate Dihydrate respiratory (inhalation) | Apr 23, 2026 | |
| EXENATIDE GLP-1 Receptor Agonist [EPC] | Byetta subcutaneous | Apr 28, 2005 | |
| ACALABRUTINIB Kinase Inhibitor [EPC] | CALQUENCE oral | — | Oct 31, 2017 |
AstraZeneca PLC Trial Locations
AstraZeneca PLC clinical trials are running at 1,163 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 (42)
Diseases targeted by AstraZeneca PLC's clinical trial and drug development programs
Adrenocortical carcinoma is a rare, aggressive cancer of the adrenal cortex, the outer layer of the adrenal glands that sit on top of each kidney. These glands produce essential hormones including cor...
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...
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...
Patient Resources
Organizations and resources related to AstraZeneca PLC's rare disease focus areas
Frequently Asked Questions About AstraZeneca PLC
Common questions about AstraZeneca PLC's rare disease programs, clinical trials, and treatments.