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Sobi North America’s Immunology portfolio addresses serious, disabling or even life-threatening diseases. With our understanding of the mechanisms involved, we are studying how our existing products can potentially help in new indications and we are searching for new late-stage treatment candidates that show promise in other areas of unmet medical need.

We specialize in treatment options for hemophagocytic lymphohistiocytosis (HLH), an extremely rare, rapidly progressive and often-fatal syndrome of hyperinflammation in which there is a massive overproduction of Interferon-gamma (IFN-gamma or IFNγ), a cytokine secreted by cells of the immune system to help regulate immune functions. This drives the immune system into hyperactivation, which ultimately leads to organ failures. The primary form of the disease is typically seen in pediatric patients and is fatal if untreated, with a median survival of less than two months. The secondary (acquired) form of the disease typically arises later in life and is also associated with significant mortality.

About 100 cases of primary HLH are diagnosed each year in the US, but this is believed to represent an under-diagnosis. HLH is difficult to diagnose because of the variability in signs and symptoms, which may include fevers, swelling of the liver and spleen, severe low red and white blood cell counts, bleeding disorders, infections, neurological symptoms, organ dysfunction and organ failure.

The immediate goal of treatment is to bring the hyperinflammation under control quickly to make the patient eligible for a hematopoietic stem-cell transplant.

We focus on elevating patient care to new standards to improve outcomes and expand treatment options by working to identify mechanisms that promote balance in the immune system with the goal of achieving curative therapies.
 

Sobi’s focus within immunology

Hemophagocytic lympho-histiocytosis (HLH)

A rare, life-threatening condition caused by an overactive, abnormal response of the immune system. In hemophagocytic lymphohistiocytosis, the immune system responds to a stimulus or ‘trigger’, often an infection, but the response is ineffective and abnormal.

 

Still´s Disease

A rare systemic autoinflammatory disease characterised by fevers, rash, and joint pain. Still’s disease includes Systemic juvenile idiopathic arthritis (SJIA) and Adult-Onset Still's disease (AOSD) which share symptoms but vary in frequency and presentation. A potentially fatal complication is macrophage activation syndrome (MAS).

Therapeutic areas

Learn more about all our therapeutic areas spanning - Immunology, Hematology and Specialty Care.

Our medicines

Read more about our medicines within rare diseases