Recombinant Human Acid Alpha-Glucosidase and Uses Thereof
AssigneeAmicus Therapeutics, Inc.
InventorsHung V. Do, Russell Gotschall, Hing Char, Jay Barth
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Abstract
Provided herein are methods of treating Pompe disease comprising administering a population of recombinant human acid a-glucosidase molecules or a pharmaceutical composition or formulation thereof, and a pharmacological chaperone.
Claims33
A method of treating Pompe disease in a subject in need thereof, comprising administering to the subject a population of recombinant human acid α-glucosidase (rhGAA) molecules, concurrently or sequentially with a pharmacological chaperone;
- wherein the rhGAA molecules comprise seven potential N-glycosylation sites;
- wherein 40%-60% of the N-glycans on the rhGAA molecules are complex type N-glycans;
- wherein the rhGAA molecules comprise at least 0.5 mol bis-mannose-6-phosphate (bis-M6P) per mol of rhGAA at the first potential N-glycosylation site as determined using liquid chromatography tandem mass spectrometry (LC-MS/MS);
- and
- wherein the method improves one or more disease outcomes the subject compared to (1) baseline, or (2) a control treatment comprising administering alglucosidase alfa and a placebo for the pharmacological chaperone.
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The method of claim 2418, wherein, compared to the control treatment, the subject's GSGC score is improved as indicated by a decrease of at least 1 point after treatment.
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The method of claim 241, wherein the method reduces the levels of at least one marker of muscle damage comprising creatine kinase (CK), and/or the method reduces the levels of at least one marker of glycogen accumulation comprising urine hexose tetrasaccharide (Hex4).
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Dependencies are inferred from the claim text.