CSL Behring
CSL Behring works on 17 rare diseases tracked on Trial Friend, including Alpha-1 Antitrypsin Deficiency, Ataxia-Telangiectasia, Chronic Graft-versus-Host Disease and 14 more, with 13 recruiting clinical trials and 2 FDA-approved rare disease drugs.
CSL Behring is a global leader in plasma-derived and recombinant therapies for rare diseases including bleeding disorders, immune deficiencies, and hereditary angioedema. Key products include Hemgenix (etranacogene dezaparvovec), the first gene therapy for hemophilia B, Idelvion (Factor IX) for hemophilia B, Haegarda (C1 esterase inhibitor) for hereditary angioedema, and Hizentra (subcutaneous immunoglobulin) for primary immunodeficiency and CIDP. CSL Behring is part of CSL Limited, which also acquired Vifor Pharma in 2022 to expand into nephrology and iron deficiency.
CSL Behring Drug Pipeline
CSL Behring has 13 active clinical trials across 4 development stages, with 13 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 CSL Behring'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.
CSL Behring Clinical Trials (13)
Active and recruiting clinical trials sponsored by CSL Behring, 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.
CSL Behring FDA-Approved Drugs (2)
Medications developed or marketed by CSL Behring 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 |
|---|---|---|---|
| GARADACIMAB | ANDEMBRY subcutaneous | Jun 16, 2025 | |
| ETRANACOGENE DEZAPARVOVEC | HEMGENIX | — |
CSL Behring Trial Locations
CSL Behring clinical trials are running at 222 sites in 26 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 (17)
Diseases targeted by CSL Behring's clinical trial and drug development programs
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...
Ataxia-telangiectasia (A-T) is a rare inherited disorder in which the ATM gene, which coordinates DNA repair, does not work. Children lose coordination from early childhood as the cerebellum degenerat...
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...
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) is an autoimmune disorder affecting the peripheral nerves, causing progressive weakness and impaired function in the legs and arms. The immune ...
Common variable immunodeficiency is a primary immunodeficiency disorder characterized by low levels of immunoglobulins and impaired antibody responses. Patients experience recurrent infections affecti...
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 CSL Behring's rare disease focus areas
Frequently Asked Questions About CSL Behring
Common questions about CSL Behring's rare disease programs, clinical trials, and treatments.