Swedish Orphan Biovitrum, known as Sobi, is an international biopharmaceutical company dedicated to developing treatments for rare diseases. Sobi’s portfolio of therapeutic products concentrates on three main areas: haematology, immunology, and specialty care. This focus allows the company to address high unmet patient needs and transform the daily lives of people living with debilitating conditions. This overview explores the specific therapeutic products Sobi offers across its core disease areas.
What is Sobi
Sobi was formed in 2010 through the merger of Swedish Orphan, which had a portfolio of orphan drugs, and Biovitrum, which specialized in product development and manufacturing. This union established a company specifically dedicated to creating and commercializing medicines for rare diseases.
The company is headquartered in Stockholm, Sweden, and has expanded its operational presence globally, serving patients across Europe, North America, the Middle East, and Asia. Sobi’s business model revolves around the idea that while individual rare diseases are uncommon, collectively they affect an estimated 400 million people worldwide. The company leverages its expertise in biotechnology and protein biochemistry to develop treatments for these patient populations.
Haematology Treatments
Haematology represents Sobi’s largest therapeutic area, with a deep history in treating haemophilia. The company offers recombinant clotting factor therapies for Hemophilia A (Factor VIII deficiency) and Hemophilia B (Factor IX deficiency). These products utilize extended half-life technology, allowing the clotting factor to remain active in the bloodstream for a longer duration.
For Hemophilia A, Sobi markets Elocta (efmoroctocog alfa) and Altuvoct (efanesoctocog alfa), which is a newer generation treatment. The extended half-life mechanism means patients require fewer intravenous infusions to maintain protective clotting factor levels. This less frequent dosing schedule significantly improves the quality of life and adherence for patients managing this chronic condition.
For Hemophilia B, the product Alprolix (eftrenonacog alfa) provides an extended half-life Factor IX therapy. Extending the time between necessary treatments helps patients maintain a higher minimum level of the clotting factor, preventing spontaneous bleeding episodes.
Sobi’s haematology portfolio also addresses other rare blood and bone marrow conditions. Doptelet (avatrombopag) treats chronic immune thrombocytopenia (ITP) in adults who have not responded adequately to previous therapies. ITP is characterized by a low platelet count, and Doptelet stimulates the production of new platelets. Additionally, the company offers Aspaveli/Empaveli (pegcetacoplan) for paroxysmal nocturnal haemoglobinuria (PNH), a rare blood disorder characterized by the destruction of red blood cells. This product targets the complement cascade, which is overactive in PNH.
Immunology and Specialty Care
Sobi maintains a strong presence in the immunology field, focusing on treatments for rare auto-inflammatory and auto-immune conditions. Kineret (anakinra) is a foundational product used to treat conditions like Cryopyrin-Associated Periodic Syndromes (CAPS) and Familial Mediterranean Fever (FMF). This drug works by blocking the activity of interleukin-1 (IL-1), a protein central to inflammation. Kineret helps to manage the debilitating symptoms associated with these chronic inflammatory diseases.
Gamifant (emapalumab) is another specialized immunology product indicated for primary hemophagocytic lymphohistiocytosis (HLH). HLH is an ultra-rare, life-threatening syndrome of severe hyperinflammation caused by an overactive immune system. Gamifant is a monoclonal antibody that targets interferon gamma, a cytokine driving the excessive immune response in HLH.
The Specialty Care division treats rare genetic and metabolic diseases. Orfadin (nitisinone) is used for treating hereditary tyrosinemia type 1 (HT-1). HT-1 is a metabolic disorder where the body cannot effectively break down the amino acid tyrosine, leading to toxic substance accumulation. Orfadin inhibits a specific enzyme in the tyrosine breakdown pathway, preventing the formation of damaging byproducts.
Understanding Rare Disease Treatment Access
Obtaining treatments for rare diseases often involves complexities regarding access and logistics. Given the specialized nature and high cost of orphan drugs, patient access frequently requires navigating intricate reimbursement processes within different national healthcare systems. Sobi works with communities and healthcare providers to streamline access through established healthcare systems.
The company implements various patient support programs designed to address these logistical and financial hurdles. These programs can include providing educational materials, offering financial support options, and assisting with the complex adherence requirements of specialized therapies. For patients with life-threatening conditions who have exhausted all approved options, Sobi maintains a process for Managed Access Programmes.
These managed access programs allow for the provision of an investigational medicine before it has received full regulatory approval, provided strict criteria are met and local laws permit it. Furthermore, Sobi supports essential services like home nursing and specialized medicine delivery programs to ensure patients receive their treatments reliably. This focus on support is crucial because the successful use of rare disease therapies depends heavily on specialized prescribing, monitoring, and sustained patient adherence.