Treatment of paroxysmal nocturnal hemoglobinuria, hemolytic anemias and disease states involving intravascular and extravascular hemolysis
AssigneeAlexion Pharmaceuticals, Inc.
InventorsV. Michael Holers, Antonio M. Risitano
Life of this patent
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Abstract
Methods for treating subjects having complement-mediated hemolytic disorders, such as paroxysmal nocturnal hemoglobinuria (PNH) and other hemolytic anemias, the method comprising administering an effective amount of a composition that inhibits the activity of the complement alternative pathway.
Claims33
A method of treating a subject with paroxysmal nocturnal hemoglobinuria (PNH) comprising administering to said subject a terminal complement inhibitor and, subsequent to administration of the terminal complement inhibitor, administering an inhibitor of the complement alternative pathway to the subject once the subject has been determined to be non-responsive or partially responsive to the terminal complement inhibitor based on an assessment of at least one hemolytic marker of the subject, wherein said inhibitor of the complement alternative pathway is a complement receptor 2 (“CR2”)-Factor H (“FH”) molecule comprising:
- a) a CR2 portion comprising a CR2 or a fragment thereof;
- and
- b) a FH portion comprising a FH or a fragment thereof,
- wherein said CR2 portion of the CR2-FH molecule is capable of binding to a CR2 ligand, wherein said FH portion of the CR2-FH molecule is capable of inhibiting complement activation of the alternative pathway.
Dependencies are inferred from the claim text.