Af­ter se­cur­ing quick OK for PNH pa­tients, Alex­ion's Ul­tomiris suc­ceeds in aHUS piv­otal study

About a month af­ter the FDA gave Alex­ion a Christ­mas present in the form of an ear­ly ap­proval for its rare blood dis­or­der drug, Ul­tomiris, for pa­tients with PNH, the drug­mak­er has re­port­ed pos­i­tive piv­otal da­ta eval­u­at­ing the mon­o­clon­al an­ti­body in a sin­gle-arm study in­volv­ing atyp­i­cal he­molyt­ic ure­mic syn­drome (aHUS) pa­tients.

The Boston-based biotech is now prep­ping reg­u­la­to­ry sub­mis­sions in the Unit­ed States, EU and Japan for Ul­tomiris. The drug is Alex­ion’s shot at pro­tect­ing its block­buster blood dis­or­der fran­chise that is cur­rent­ly cen­tered around its flag­ship drug, Soliris, which is a com­ple­ment in­hibitor typ­i­cal­ly ad­min­is­tered every two weeks. Ul­tomiris has a sim­i­lar mech­a­nism of ac­tion but re­quires less-fre­quent dos­ing — every eight weeks.

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