Alexion advances on ULTOMIRIS®, amyloidosis

Alexion reports that the Committee for Medicinal Products for Human Use has adopted a positive opinion, recommending marketing authorization in the European Union for a new advanced formulation of ULTOMIRIS® (ravulizumab). ULTOMIRIS is the first and only C5 inhibitor treatment for two ultra-rare diseases — paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome … more

In addition, Japan has approved ULTOMIRIS® for adults and children living with atypical hemolytic uremic syndrome … more

Separately, Alexion and Caelum Biosciences have initiated a program to evaluate CAEL-101, a first-in-class amyloid fibril targeted therapy, in combination with standard-of-care therapy in AL amyloidosis … more