Sobi is committed to revolutionizing the treatment and care for people living with rare and severe diseases. Our medicines are concentrated within the areas of hematology, immunology and specialty care.
Hematology
At Sobi North America, we’re advancing blood cancer treatments that have life-changing potential.
We are committed to delivering treatment options for patients with Immune thrombocytopenia (ITP), a rare autoimmune disease affecting platelet production and characterized by fatigue, bruising and bleeding. Only nine or ten people in every 100,000 have ITP. Until recently, treatment options are ruled by compromise. Today, there is increased awareness of the available treatment options, and better life may be possible for people living with ITP.
Thrombocytopenia is a deficiency of thrombocytes, or platelets. Platelets are tiny, colorless cell fragments in the blood that form clots, which stop or prevent bleeding.
Thrombocytopenia that is not caused by a previous disease is known as primary ITP. If the disease is caused by another condition affecting the immune system (such as HIV), it is referred to as secondary.
Our portfolio also addresses cytopenic myelofibrosis, a rare blood disease characterized by scarring of the bone marrow, as well as disruption of the normal production of blood cells. Approximately 20,000 patients in the US are diagnosed with myelofibrosis, and about one-third of those have severe thrombocytopenia, with a median overall survival of 15 months.
We are dedicated to delivering the promise of a better life for those living with rare and severe blood diseases.
Immunology
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.
Specialty Care
We are dedicated to treatment options for respiratory syncytial virus (RSV), a common and highly contagious seasonal virus that is contracted by nearly all infants by the age of two. In most babies, RSV causes only a mild respiratory infection; however, for some – especially babies who are considered high risk – RSV can become much more serious. RSV is the leading cause of hospital admission in infants aged less than one year in the US.