Octapharma
Octapharma works on 9 rare diseases tracked on Trial Friend, including Alpha-1 Antitrypsin Deficiency, Chronic Inflammatory Demyelinating Polyneuropathy, Common Variable Immunodeficiency and 6 more, with 9 active clinical trials (8 recruiting).
Octapharma is a family-owned Swiss plasma-medicine specialist headquartered in Lachen and founded in 1983, and it describes itself as the largest privately owned and independent plasma fractionator in the world. Its business is turning donated human plasma into therapies for rare bleeding and immune disorders, an old-fashioned model it has run at global scale for 4 decades, alongside recombinant products grown from human cell lines.
For bleeding-disorder patients, its mainstays are Nuwiq, a recombinant factor VIII for hemophilia A, and wilate, a von Willebrand factor/factor VIII concentrate whose FDA approval expanded in July 2026 to cover routine prophylaxis in children under 6 with von Willebrand disease. In immunology, its immunoglobulin therapies include Panzyga, approved for adults with chronic inflammatory demyelinating polyneuropathy (CIDP). Its current trials include a Phase 3 study of Panzyga in children with CIDP and a Phase 3 study of a plasma-derived C1 esterase inhibitor for hereditary angioedema attacks.
Octapharma rarely makes biotech headlines because it has no stock ticker and no splashy platform story. What it has is durability: plasma collection centers, manufacturing depth, and a portfolio of unglamorous products that thousands of rare disease patients depend on every week.
Octapharma Drug Pipeline
Octapharma has 9 active clinical trials across 4 development stages, with 8 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.
Octapharma Clinical Trials (9)
Active and recruiting clinical trials sponsored by Octapharma, 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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FDA-Approved Drugs
Octapharma Trial Locations
Octapharma clinical trials are running at 153 sites in 34 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 (9)
Diseases targeted by Octapharma'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 protein alpha-1 antitrypsin. Without adequate protection, neutro...
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...
Dermatomyositis is a rare autoimmune inflammatory disease causing muscle weakness and distinctive skin rashes, particularly over joints. It involves inflammation of muscles and skin blood vessels. Abo...
Hemophilia A is an X-linked bleeding disorder caused by deficiency or dysfunction of clotting factor VIII. Severity depends on factor levels, ranging from mild to severe hemorrhage. Modern factor repl...
Hemophilia B is an X-linked bleeding disorder caused by deficiency or dysfunction of clotting factor IX. Clinically indistinguishable from Hemophilia A, the key difference is factor type. Modern facto...
Patient Resources
Organizations and resources related to Octapharma's rare disease focus areas
Frequently Asked Questions About Octapharma
Common questions about Octapharma's rare disease programs, clinical trials, and treatments.