PNH is a ultra-rare, life-threatening blood disorder in which the immune system destroys red blood cells.
The benefits of orphan drug status in the EU include a 10-year period of market exclusivity for the indication, if approved.
ALXN1210, which is not approved in any country, is being assessed in a phase 1/2 study and a phase 2 study in patients with PNH. Target enrollment has been exceeded in both trails.
The company also plans to start a clinical program with ALXN1210 later this year in patients with atypical hemolytic uremic syndrome, another ultra-rare and life-threatening disease caused by chronic uncontrolled complement activation.
In early trials, ALXN1210 showed rapid, complete, and sustained reduction of free C5 activity and a terminal half-life of over 30 days, which may facilitate a monthly or longer dosing interval.
ALXN1210 will replace Soliris (eculizumab), approved in the European Union in June 2007. It has a half-life nearly three times that of Soliris.
PNH occurs in people of all ages, with an average age of onset in the early 30s. It has been identified more commonly among patients with disorders of the bone marrow, including aplastic anemia and myelodysplastic syndromes.