ASH: First-in-class rare disease med performs well in extension study

Britain’s largest drugmaker, AstraZeneca (LSE: AZN), has announced positive long-term results from the Phase III ALPHA trial at the annual meeting of the American Society of Hematology (ASH).

The trial evaluates the impact of adding danicopan to treatment with Ultomiris (ravulizumab) or Soliris (eculizumab), for people with the rare disease paroxysmal nocturnal hemoglobinuria (PNH).

The investigational small molecule offers the potential for a new mechanism of action to help people with the rare blood disorder, a debilitating condition where the immune system attacks and damages red blood cells.