Patent SG-11201804965S-A (SG)
Title: Augmented acid alpha-glucosidase for the treatment of pompe disease
Inventor: DO HUNG V
IPC: A61K38/47
Filed: 2016-12-29
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization 111111110111101110101011111010111110111011101011101110111011111111101111011111 International Bureau 0.. .... .. (10) International Publication Number (43) International Publication Date ..... ...r .....1 WO 2017/117407 Al 6 July 2017 (06.07.2017) WI P0 I P CT (51) International Patent Classification: (81) Designated States (unless otherwise indicated, for every A61K 38/47 (2006.01) A61K 31/445 (2006.01) kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, (21) International Application Number: BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, PCT/US2016/069243 DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, (22) International Filing Date: HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, 29 December 2016 (29.12.2016) KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, (25) Filing Language: English NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, (26) Publication Language: English RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, (30) Priority Data: ZA, ZM, ZW. 62/272,890 30 December 2015 (30.12.2015) US 62/300,479 26 February 2016 (26.02.2016) US (84) Designated States (unless otherwise indicated, for every 62/315,412 30 March 2016 (30.03.2016) US kind of regional protection available): ARIPO (BW, GH, 62/402,454 30 September 2016 (30.09.2016) US GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, 62/428,867 1 December 2016 (01.12.2016) US TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, 62/431,791 8 December 2016 (08.12.2016) US TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, 15/394,135 29 December 2016 (29.12.2016) US DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, (71) Applicant: AMICUS THERAPEUTICS, INC. [US/US]; SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, 1 Cedar Brook Drive, Cranbury, New Jersey 08512 (US). GW, KM, ML, MR, NE, SN, TD, TG). (72) Inventors: DO, Hung V.; 1 Cedarbrook Drive, Cranbury, Published: New Jersey 08512 (US). KHANNA, Richie; 1 Cedar — with international search report (Art. 21(3)) Brook Drive, Cranbury, New Jersey 08512 (US). GOTSCHALL, Russell; 1 Cedar Brook Drive, Cranbury, — with sequence listing part of description (Rule 5.2(a)) New Jersey 08512 (US). Agent: ALEGRIA, Rory P.; Servilla Whitney LLC, 33 (74) Wood Avenue South, Suite 830, Iselin, New Jersey 08830 (US). 1-1 IN © 7r IN 11 11 IN 1-1 (54) Title: AUGMENTED ACID ALPHA-GLUCOSIDASE FOR THE TREATMENT OF POMPE DISEASE 0 N (57) : A method for treating Pompe disease including administration of recombinant human acid a-glucosidase having op - en. ) timal glycosylation with mannose-6-phosphate residues in combination with an amount of miglustat effective to maximize tissue up- take of recombinant human acid a-glucosidase while minimizing inhibition of the enzymatic activity of the recombinant human acid a-glucosidase is provided.