Orpha Biotech develops innovative solutions for the treatment of diseases that occur at rare frequency and for which effective therapies currently do not exist.
Such diseases are often referred to as orphan diseases, since their low incidence has meant their market size has been too small for mainstream drug developers to place resources behind them, leaving the patient population in effect neglected and orphaned.
A rare disease is defined in Europe as any life-threatening or chronically debilitating disease that affects fewer than 5 in 10,000 people. Thousands of rare diseases have been identified and the cumulative population prevalence of rare diseases is estimated to be 3.5-5.9% globally at any given time. In other words, while the incidence of each rare disease is by definition quite low, the overall number of people across the globe affected by rare diseases is substantial.