Ablynx, the llama antibody biotech in Belgium, has now proven to be on solid ground regarding their orphan drug for a rare blood disease. Now the New England Journal of Medicine has published their phase II results.
Despite being actively developing therapies in partnerships with big pharma for different diseases (including multiple sclerosis with Genzyme, cancer with MSD, and with NovoNordisk in a yet undisclosed disease), their flagship is a drug for the rare acquired thrombotic thrombocytopenic purpura (aTTP).
This blood disorder is an autoimmune disease which afflicts only about 5 people in a million per year, but can lead to severe complications (like brain damage and kidney failure). It is currently treated with a daily plasma exchange (TPE) – a sort of blood “cleaning”, that requires people to stay in the hospital for long periods of time,