# US 10,245,304 B2 · Treatment of alpha-galactosidase a deficiency > Agents: this data is available as JSON from the patentgrep API and MCP server — see https://patentgrep.com/llms.txt - Number: US 10,245,304 B2 - Document ID: US10245304B2 - Status: Granted - Kind: B2 - Application number: 15/350,785 - Filed: 2016-11-14 - Published: 2017-08-03 - Granted: 2019-04-02 - Earliest claimed: 2002-04-25 - Assignees: Shire Human Genetic Therapies, Inc. - Applicants: Shire Human Genetic Therapies, Inc. - Inventors: Douglas A. Treco; Kenneth Loveday; Marianne Borowski - Web page: https://patentgrep.com/patent/US10245304B2 ## CPC - A61K 38/47: acting on glycosyl compounds (3.2), e.g. cellulases, lactases - A61K 38/00: Medicinal preparations containing peptides (peptides containing beta-lactam rings A61K31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K31/00; ergot alkaloids of the cyclic peptide type A61K31/48; containing macromolecular compounds having statistically distributed amino acid units A61K31/74; medicinal preparations containing antigens or antibodies A61K39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K47/00) - A61P 3/00: Drugs for disorders of the metabolism (of the blood or the extracellular fluid A61P7/00) - A61P 3/06: Antihyperlipidemics - A61P 7/00: Drugs for disorders of the blood or the extracellular fluid - A61P 9/00: Drugs for disorders of the cardiovascular system - A61P 13/12: of the kidneys - A61P 25/00: Drugs for disorders of the nervous system - A61P 25/02: for peripheral neuropathies - A61P 25/04: Centrally acting analgesics, e.g. opioids - A61P 39/02: Antidotes - A61P 43/00: Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 - C12Q 1/54: involving glucose or galactose - C12Y 302/01022: Alpha-galactosidase (3.2.1.22) - G01N 2400/10: Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters - A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D) - A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS - C12Q: MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS (immunoassay G01N33/53); COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES - C12Y: ENZYMES - G01N: INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES (measuring or testing apparatus or processes other than immunoassay, involving enzymes or microorganisms C12M, C12Q) ## Abstract The invention provides methods of treating α-galactosidase A deficiency. Dosage forms, methods of administration, and methods of analyzing human α-galactosidase A are also included. ## Claims ### Claim 1 A kit for the treatment of α-Gal A deficiency, comprising: (a) a human α-Gal A glycoprotein preparation; and (b) instructions to administer the preparation to a subject in need thereof, wherein the serum clearance of the α-Gal A preparation from subject's circulation is less than 4 mL/min/kg on the linear portion of the AUC vs. dose curve, wherein the α-Gal A glycoprotein preparation has the following four characteristics: (1) has less than 35% tri- and tetra-sialylated glycans combined; (2) has greater than 50% complex glycans; (3) has less than 45% phosphorylated glycans; and (4) has a ratio of sialylated glycans to phosphorylated glycans greater than 1. ### Claim 2 (depends on 1) The kit of claim 1, wherein the serum clearance of the α-Gal A preparation from subject's circulation is 3.5 mL/min/kg or less on the linear portion of the AUC vs. dose curve. ### Claim 3 (depends on 1) The kit of claim 1, wherein the serum clearance of the α-Gal A preparation from subject's circulation is 3.0 mL/min/kg or less on the linear portion of the AUC vs. dose curve. ### Claim 4 (depends on 1) The kit of claim 1, further comprising instructions to administer a unit dose of the α-Gal A preparation of between 0.05 mg and 2.0 mg per kilogram of body weight. ### Claim 5 (depends on 4) The kit of claim 4, wherein the instructions to administer the unit dose of the α-Gal A preparation specify that the unit dose is between 0.05 mg and 1.0 mg per kilogram of body weight. ### Claim 6 (depends on 4) The kit of claim 4, wherein the instructions to administer the unit dose of the α-Gal A preparation specify that the unit dose is between 0.05 mg and 0.5 mg per kilogram of body weight. ### Claim 7 (depends on 4) The kit of claim 4, further comprising instructions to administer the unit dose no more than once every 8 weeks. ### Claim 8 (depends on 4) The kit of claim 4, further comprising instructions to administer the unit dose no more than once every 6 weeks. ### Claim 9 (depends on 4) The kit of claim 4, further comprising instructions to administer the unit dose no more than once every 4 weeks. ### Claim 10 A kit for the treatment of α-Gal A deficiency, comprising: a human α-Gal A glycoprotein preparation and instructions to administer the preparation to a subject in need thereof at a unit dose of less than 1.0 mg per kilogram of body weight of the subject, wherein the α-Gal A glycoprotein preparation has the following four characteristics: (1) has less than 35% tri- and tetra-sialylated glycans combined; (2) has greater than 50% complex glycans; (3) has less than 45% phosphorylated glycans; and (4) has a ratio of sialylated glycans to phosphorylated glycans greater than 1. ### Claim 11 (depends on 10) The kit of claim 10, wherein the instructions to administer the unit dose specify that the unit dose is less than 0.5 mg per kilogram of body weight of the subject. ### Claim 12 (depends on 10) The kit of claim 10, further comprising instructions to administer the unit dose no more than once every 8 weeks. ### Claim 13 (depends on 12) The kit of claim 12, wherein the kit comprises instructions to administer the unit dose no more than once every 6 weeks. ### Claim 14 (depends on 12) The kit of claim 12, wherein the kit comprises instructions to administer the unit dose no more than once every 4 weeks. ### Claim 15 A kit for the treatment of α-Gal A deficiency, comprising a human α-Gal A glycoprotein preparation and instructions to administer the preparation to a subject in need thereof no more than once every 8 weeks, wherein the α-Gal A glycoprotein preparation has the following four characteristics: (1) has less than 35% tri- and tetra-sialylated glycans combined; (2) has greater than 50% complex glycans; (3) has less than 45% phosphorylated glycans; and (4) has a ratio of sialylated glycans to phosphorylated glycans greater than 1. ### Claim 16 (depends on 15) The kit of claim 15, wherein the instructions comprise instructions to administer the preparation to a subject in need thereof no more than once every 6 weeks. ### Claim 17 (depends on 15) The kit of claim 15, wherein the instructions comprise instructions to administer the preparation to a subject in need thereof no more than once every 4 weeks. ### Claim 18 (depends on 15) The kit of claim 15, further comprising instructions to administer a unit dose of the α-Gal A preparation of between 0.05 mg and 2.0 mg per kilogram of body weight. ### Claim 19 (depends on 18) The kit of claim 18, wherein the instructions to administer the unit dose of the α-Gal A preparation specify that the unit dose is between 0.05 and 1.0 mg per kilogram of body weight. ### Claim 20 (depends on 18) The kit of claim 18, wherein the instructions to administer the unit dose of the α-Gal A preparation specify that the unit dose is between 0.05 and 0.5 mg per kilogram of body weight.