Alexion adds fourth indication for C5 inhibitor Ultomiris in rare autoimmune disorder

As­traZeneca’s rare dis­ease arm Alex­ion scored its fourth ap­proval for Ul­tomiris in a rare au­toim­mune dis­or­der af­ter Phase 3 da­ta showed there were ze­ro pa­tients with re­laps­es over 73 weeks.

The FDA ap­proved Ul­tomiris, the first and on­ly long-act­ing C5 com­ple­ment in­hibitor, for the treat­ment of adult pa­tients with an­ti-aqua­por­in-4 an­ti­body-pos­i­tive neu­romyelitis op­ti­ca spec­trum dis­or­der (NMOSD), a rare au­toim­mune dis­ease that af­fects the cen­tral ner­vous sys­tem and can re­lapse un­pre­dictably. Symp­toms can in­clude blind­ness and neu­ro­log­i­cal prob­lems that can lead to per­ma­nent dis­abil­i­ties.

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