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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