Alexion Pharmaceuticals, Inc.
METHODS FOR REDUCING LIVER FIBROSIS AND TREATING LYSOSOMAL ACID LIPASE DEFICIENCY IN PATIENTS BASED ON ISHAK FIBROSIS STAGE

Abstract:

The present invention provides methods of reducing liver fibrosis in a human patient with a lysosomal acid lipase (LAL) deficiency comprising administering sebelipase alfa to the patient, wherein the patient has been determined to have at least a one point reduction (e.g., a .gtoreq.1 point reduction or a .gtoreq.2 point reduction) in Ishak fibrosis stage after administration compared to a baseline Ishak fibrosis stage obtained from the patient prior to administration. Also provided are methods of treating a human patient with a lysosomal acid lipase (LAL) deficiency comprising administering sebelipase alfa to the patient, wherein the patient has been determined to have at least a one point reduction (e.g., a .gtoreq.1 point reduction or a .gtoreq.2 point reduction) in Ishak fibrosis stage after administration compared to a baseline Ishak fibrosis stage obtained from the patient prior to administration.