Sobi at a glance
At Sobi, we are transforming life for people with rare diseases. As an international biopharmaceutical company, we provide access to innovative treatments in the areas of haematology, immunology and specialty care.
- Two therapeutic areas – Haematology and Immunology – and a portfolio of products within Specialty Care
- Our extended half-life factor replacement treatments are the most prescribed treatments for haemophilia A and B respectively in several markets
- A strong portfolio of on-market products, and an extensive portfolio of pre-market assets
- An international biopharmaceutical company covering the entire value chain
- We put our focus where we can have the greatest impact, in late-stage research and development, and in patient access and commercialisation
- Revenue of SEK 15.3 bn for the full year 2020
- Global Head Office in Stockholm, with offices in more than 30 countries, delivering treatments to patients in more than 70 countries
- Approximately 1,500 employees globally
There are thought to be around 6,000 distinct rare diseases, affecting more than 300 million people around the world. Yet around 95 per cent of rare diseases currently have no approved treatment.
95% of rare diseases currently have no approved treatment
300 million people affected
75% of rare diseases affect children
The rare disease space is unlike any other in medicine. In Europe, a rare disease is defined as one affecting fewer than one person per 2,000. In the US, the Orphan Drug Act of 1983 defines a rare disease as a condition affecting fewer than 200,000 people.
High unmet medical need
An estimated 75 per cent of identified rare diseases affect children, often having a devastating effect on life expectancy and quality of life. Around 30 per cent of children diagnosed with a rare disease will not live to see their fifth birthday. The severe nature of many rare diseases often leads to high levels of distress for patients and their families, with an accompanying high treatment burden.
Around 80 per cent of rare diseases are inherited rather than acquired: they involve a defect in the genes that tell our bodies how to work. As a result, the body may fail to produce an essential enzyme or protein, for example, or its own immune defences may attack its own systems.
Many physicians may never have seen a specific rare condition before; as a result, many cases can go undiagnosed for years.
The rare disease landscape presents specific scientific, medical and commercial challenges. They range from understanding the biology of a disease, identifying molecules that can become successful treatments, developing the complex biopharmaceutical processes to manufacture a drug, designing and running clinical trials in extremely small patient populations, obtaining regulatory approvals, negotiating pricing and reimbursement, through to working with healthcare professionals and patients to ensure access for the people who need the treatment.
Such treatments are referred to as “orphan drugs,” intended to treat diseases so rare that companies would be reluctant to develop them under normal market conditions.
Our therapies are concentrated within the areas of haematology, immunology and specialty care.
Within haematology we provide extended half-life recombinant replacement clotting factor treatments for haemophilia A and haemophilia B, and a treatment for thrombocytopenia, a condition characterised by abnormally low levels of platelets in the blood.
The field of immunology has long been at the heart of what we do at Sobi, allowing us to gain extensive experience over many years. Our Immunology portfolio allows the treatment of serious, disabling or even life-threatening diseases.
We also provide important specialty treatments in the area of genetics and metabolism.
The area of haematology covers a wide range of conditions, including those involving red and white blood cells, coagulation and platelets, and bone marrow.
The field of immunology has long been at the heart of what we do at Sobi, allowing us to gain extensive experience over many years.
We provide products for the treatment of genetic and metabolic diseases as well as a number of specialist indications.
Research and development
We bring new treatments to market, expand our existing products into new indications and regions where they address unmet medical need, and gather real-world evidence for available products.
Our R&D is focused on late-stage research within the areas of Haematology and Immunology, where our pre-market assets represent considerable value.