| EP2932982 - A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives [Right-click to bookmark this link] | Status | No opposition filed within time limit Status updated on 09.08.2019 Database last updated on 08.04.2026 | |
| Former | The patent has been granted Status updated on 31.08.2018 | ||
| Former | Grant of patent is intended Status updated on 11.04.2018 | ||
| Former | Examination is in progress Status updated on 25.11.2016 | Most recent event Tooltip | 09.07.2021 | Lapse of the patent in a contracting state New state(s): HU | published on 11.08.2021 [2021/32] | Applicant(s) | For all designated states Amicus Therapeutics, Inc. 1 Cedar Brook Drive Cranbury, NJ 08512 / US | [2015/43] | Inventor(s) | 01 /
Mugrage, Benjamin 1 Cedar Brook Drive Cranbury, NJ 08512 / US | [2015/43] | Representative(s) | Miller Sturt Kenyon 9 John Street London WC1N 2ES / GB | [2015/43] | Application number, filing date | 15153174.6 | 17.05.2006 | [2015/43] | Priority number, date | US20050682241P | 17.05.2005 Original published format: US 682241 P | US20050729329P | 21.10.2005 Original published format: US 729329 P | [2015/43] | Filing language | EN | Procedural language | EN | Publication | Type: | A1 Application with search report | No.: | EP2932982 | Date: | 21.10.2015 | Language: | EN | [2015/43] | Type: | B1 Patent specification | No.: | EP2932982 | Date: | 03.10.2018 | Language: | EN | [2018/40] | Search report(s) | (Supplementary) European search report - dispatched on: | EP | 16.07.2015 | Classification | IPC: | A61K48/00 | [2015/43] | CPC: |
A61K31/445 (EP,KR,US);
A61K31/44 (US);
A61K31/4525 (EP,US);
A61K31/4545 (EP,US);
A61K38/47 (EP,US);
A61K9/20 (KR);
A61P1/16 (EP);
A61P11/04 (EP);
A61P21/00 (EP);
A61P25/18 (EP);
A61P3/00 (EP,US);
A61P43/00 (EP);
A61P9/04 (EP);
C07D211/46 (EP,US);
C07D405/06 (EP,US);
C07D409/06 (EP,US)
(-)
| Designated contracting states | AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HU, IE, IS, IT, LI, LT, LU, LV, MC, NL, PL, PT, RO, SE, SI, SK, TR [2015/43] | Title | German: | Verfahren zur Behandlung der Pompe-Krankheit mit 1-desoxynojirimycin und Derivaten | [2015/43] | English: | A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives | [2015/43] | French: | Procédé pour le traitement de la maladie de Pompe au moyen de 1-désoxynojirimycine et de ses dérivés | [2015/43] | Examination procedure | 27.02.2015 | Examination requested [2015/43] | 21.04.2016 | Amendment by applicant (claims and/or description) | 23.11.2016 | Despatch of a communication from the examining division (Time limit: M04) | 24.03.2017 | Reply to a communication from the examining division | 09.08.2017 | Despatch of a communication from the examining division (Time limit: M02) | 16.10.2017 | Reply to a communication from the examining division | 12.04.2018 | Communication of intention to grant the patent | 14.08.2018 | Receipt of the translation of the claim(s) | 22.08.2018 | Fee for grant paid | 22.08.2018 | Fee for publishing/printing paid | Parent application(s) Tooltip | EP06770637.4 / EP1896083 | Divisional application(s) | EP18190283.4 / EP3441090 | EP20177473.4 / EP3782655 | Opposition(s) | 04.07.2019 | No opposition filed within time limit [2019/37] | Fees paid | Renewal fee | 27.02.2015 | Renewal fee patent year 03 | 27.02.2015 | Renewal fee patent year 04 | 27.02.2015 | Renewal fee patent year 05 | 27.02.2015 | Renewal fee patent year 06 | 27.02.2015 | Renewal fee patent year 07 | 27.02.2015 | Renewal fee patent year 08 | 27.02.2015 | Renewal fee patent year 09 | 12.05.2015 | Renewal fee patent year 10 | 10.05.2016 | Renewal fee patent year 11 | 10.05.2017 | Renewal fee patent year 12 | 11.05.2018 | Renewal fee patent year 13 |
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| Responsibility for the accuracy, completeness or quality of the data displayed under the link provided lies entirely with the Unified Patent Court. | Lapses during opposition Tooltip | HU | 17.05.2006 | AT | 03.10.2018 | CY | 03.10.2018 | CZ | 03.10.2018 | DK | 03.10.2018 | EE | 03.10.2018 | FI | 03.10.2018 | LT | 03.10.2018 | LV | 03.10.2018 | MC | 03.10.2018 | NL | 03.10.2018 | PL | 03.10.2018 | RO | 03.10.2018 | SE | 03.10.2018 | SI | 03.10.2018 | SK | 03.10.2018 | TR | 03.10.2018 | BG | 03.01.2019 | GR | 04.01.2019 | IS | 03.02.2019 | PT | 03.02.2019 | IE | 17.05.2019 | LU | 17.05.2019 | BE | 31.05.2019 | CH | 31.05.2019 | LI | 31.05.2019 | [2021/32] |
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| Former [2019/17] | NL | 03.10.2018 | Documents cited: | Search | [Y] WO2004069190 (SINAI SCHOOL MEDICINE et al.) | [Y] US6583158 (FAN JIAN-QIANG et al.) | [Y] WO0197829 (GENZYME CORP et al.) | [A] US2004204379 (CHENG SENG H et al.) | [A] MELLOR H R ET AL: "Cellular effects of deoxynojirimycin analogues: uptake, retention and inhibition of glycosphingolipid biosynthesis", BIOCHEMICAL JOURNAL, vol. 381, no. Part 3, 1 August 2004 (2004-08-01), pages 861 - 866, XP002541351, ISSN: 0264-6021 | [AP] PARENTI G ET AL: "Alpha-glucosidase enhancement in fibroblasts from patients with Pompe disease", JOURNAL OF INHERITED METABOLIC DISEASE, KLUWER ACADEMIC PUBLISHERS, DO, vol. 28, no. suppl.1, 1 July 2005 (2005-07-01), Abstracts - SSIEM 42st Annual Symposium, Paris, France, 6-9 September, 2005, pages 193, XP002541352, ISSN: 1573-2665 | [AP] BUTTERS T D ET AL: "Imino Sugar Inhibitors for Treating the lysosomal Glycosphingolipidoses", GLYCOBIOLOGY, OXFORD UNIVERSITY PRESS, US, vol. 15, no. 10, 1 October 2005 (2005-10-01), pages 43R - 52R, XP008124586, ISSN: 0959-6658, [retrieved on 20050518], DOI: 10.1093/GLYCOB/CWI076 DOI: http://dx.doi.org/10.1093/GLYCOB/CWI076 | by applicant | US6537785 | US5798366 | US6291657 | US6660749 | US6274597 | US6583158 | US6589964 | US6599919 | US6916829 | US98842804 | US98842704 | US5900360 | US6270954 | US6541195 | US4806650 | EP0049858 | WO2005063706 | US4639436 | WO2004037373 | WO9522975 | US5399567 | US5310745 | WO0056713 | US4880917 | EP0315017 | US5051407 | US4266025 | US5695969 | US4246345 | US430307 | US4407957 | US5798113 | US5654007 | US5780014 | US5814607 | US5879327 | US5520639 | US5846233 | US5704911 | US5580757 | US6395884 | US6458574 | US6461609 | US6210666 | US6083725 | US6451600 | US5236838 | US5879680 | US6188045 | US9721496 | US5672344 | US5139941 | US5252479 | US5753500 | WO9709441 | US5670488 | US6244113 | US6200812 | US4980286 | US5635493 | WO9206180 | WO9222635 | WO9220316 | WO9314188 | US5939401 | US6331524 | US5283185 | US5767099 | WO9309222 | BEUTLER ET AL.: "The Metabolic and Molecular Bases of Inherited Disease, 8th ed", 2001, MCGRAW-HILL, pages: 3635 - 3668 | SCRIVER, C. R., BEAUDET, A. L., SLY, W. S. AND VALLE, D.,: "The Metabolic and Molecular Bases oflnherited Disease", 1995, MCGRAW-HILL, pages: 2529 - 2561 | WEISMANN ET AL., SCIENCE, vol. 169, 1970, pages 72 - 74 | SCRIVER ET AL.: "The Metabolic and Molecular Bases of Inherited Disease 8th ed.", 2001, MCGRAW-HILL, pages: 3589 - 3610 | KLINGE ET AL., NEUROPEDIATRICS, vol. 36, no. 1, 2005, pages 6 - 11 | RABEN ET AL., MOL THER., vol. 11, no. 1, 2005, pages 48 - 56 | ISHII ET AL., BIOCHEM. BIOPHYS. RES. COMM., vol. 220, 1996, pages 812 - 815 | FAN ET AL., NATURE MED, vol. 5, 1999, pages 112 - 115 | MONTALVO ET AL., MOL GENET METAB., vol. 81, no. 3, 2004, pages 203 - 8 | HERMANS ET AL., HUMAN MUTATION, vol. 23, 2004, pages 47 - 56 | WELCH ET AL., CELL STRESS AND CHAPERONES, vol. 1, no. 2, 1996, pages 109 - 115 | WELCH ET AL., JOURNAL OF BIOENERGETICS AND BIOMEN7BRANES, vol. 29, no. 5, 1997, pages 491 - 502 | HERMANS ET AL., J BIOL. CHEM., vol. 266, 1991, pages 13507 - 12 | LIN ET AL., ZHONGHUA MIN GUO XIAO ER KE YI XUE HUI ZA ZHI., vol. 37, no. 2, 1996, pages 115 - 21 | LIN ET AL., BIOCHEM BIOPHYS RES COMMUN, vol. 208, no. 2, 17 December 1994 (1994-12-17), pages 886 - 93 | NEW ET AL., PEDIATR NEUROL., vol. 29, no. 4, 2003, pages 284 - 7 | KROOS ET AL., NEUROMUSCUL DISORD., vol. 14, no. 6, 2004, pages 371 - 4 | FERNANDEZ-HOJAS ET AL., NEUROMUSCUL DISORD., vol. 12, no. 2, 2002, pages 159 - 66 | KROOS ET AL., CLIN GENET., vol. 53, no. 5, 1998, pages 379 - 82 | HUIE ET AL., BIOCHEM BIOPHYS RES COMMUN., vol. 244, no. 3, 27 December 1996 (1996-12-27), pages 921 - 7 | HERMANS ET AL., HUM MOL GENET., vol. 3, 1994, pages 2213 - 8 | HUIE ET AL., HUM MOL GENET., vol. 3, no. 7, 1994, pages 1081 - 7 | HERMANS ET AL., HUM MUTAT., vol. 2, no. 4, 1993, pages 268 - 73 | ZHONG ET AL., AM J HUM GENET., vol. 49, no. 3, 1991, pages 635 - 45 | HERMANS ET AL., BIOCHEM BIOPHVS RES COMMUN., vol. 179, no. 2, 1991, pages 919 - 26 | RABEN ET AL., HUM MUTAT., vol. 13, no. 1, 1999, pages 83 - 4 | E.W. MARTIN: "Remington's Pharmaceutical Sciences, 18th Edition", MACK PUBLISHING | ARAI M ET AL., CIRCULATION, vol. 97, no. 13, 7 April 1998 (1998-04-07), pages 1290 - 7 | SAWLCAR AR ET AL., PROC NATL ACAD SCI USA., vol. 99, no. 24, 26 December 2001 (2001-12-26), pages 15428 - 33 | DRENT ET AL., DIABETES NUTR METAB., vol. 15, no. 3, 2002, pages 152 - 9 | DE LUIS ROMAN DA, REV CLIN ESP., vol. 204, no. 1, January 2004 (2004-01-01), pages 32 - 4 | BERNOTAS RC ET AL., BIOCHEM J, vol. 270, no. 2, 1 September 1990 (1990-09-01), pages 539 - 40 | BOLS ET AL., JOURNAL OF CARBOHYDRATE CHEMISTRY, vol. 23, no. 4, 2004, pages 223 - 238 | SAWKER ET AL., CHEMISTRY AND BIOLOGY, vol. 12, 2005, pages 1235 - 1244 | OVERKLEEF, JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 273, no. 41, 2005, pages 26522 - 26527 | TAN ET AL., JOURNAL OF BILOGICAL CHEMISTRY, vol. 266, no. 22, 1991, pages 14504 - 14510 | ROMANIOUK ET AL., GLYCOBIOLOGY, vol. 14, no. 4, 2004, pages 301 - 310 | LESUR, BIOORGANIC AND MEDICINAL CHEMISTRY LETTERS, vol. 7, no. 3, 1997, pages 355 - 360 | YOSHILCUNI, AGRIC. BIOL. CHEM, vol. 52, no. 1, 1998, pages 121 | ANZEVENO ET AL., J ORG. CHEM., vol. 54, no. 11, 1989, pages 2539 | BOSHAGEN ET AL., ANGEWANTE CHEMIE, INT. ED. ENGL., vol. 20, no. 9, 1981, pages 806 - 807 | DAVIS: "Angewante Chemie", INT. ED. ENGL., vol. 42, 2003, pages 3788 - 3792 | PERRINE ET AL., JORG. CHEM., vol. 32, 1967, pages 664 | MATOS; LOPES; LOPES, SYNTHESIS, vol. 4, 1999, pages 571 | RAO; PERLIN, CAN. J CHEM., vol. 59, 1981, pages 333 | HOOS; NAUGHTON; VASSELLA, HELV. CHIM. ACTA, vol. 76, 1993, pages 1802 | BAXTER; REITZ, J ORG. CHEM., vol. 59, 1994, pages 3175 | KINAST; SCHEDEL, ANGEW. CHEM. INT. ED. ENGL., vol. 20, 1981, pages 805 | "Current Protocols in Cell Biology", 2001, JOHN WILEY & SONS | WATSON ET AL., ADV DRUG DELIV REV, vol. 57, no. 1, 2005, pages 43 - 61 | MIYASHITA ET AL., METHODS MOL BIOL., vol. 261, 2004, pages 399 - 410 | VUKOJEVIC ET AL., CELL MOL LIFE SCI, vol. 62, no. 5, 2005, pages 535 - 50 | CHERRY ET AL., BIOCHEM SOC TRANS, vol. 31, 2003, pages 1028 - 31 | HARIGUCHI, CELL STRUCT FUNCT, vol. 27, no. 5, 2002, pages 333 - 4 | PERIASAMY, JBIOMED OPT, vol. 6, no. 3, 2001, pages 287 - 91 | OKUMIYA ET AL., MOL GENET METAB., vol. 88, no. 1, May 2006 (2006-05-01), pages 22 - 8 | LOANNU ET AL., M. J HUM. GENET., vol. 68, 2001, pages 14 - 25 | KLINGE ET AL., NEUROMUSCUL DISORD., vol. 15, no. 1, 2005, pages 24 - 31 | VAN DEN HOUT ET AL., J INHERIT METAB DIS., vol. 24, no. 2, 2001, pages 266 - 74 | AMALFITANO ET AL., GENET MED., vol. 3, no. 2, 2001, pages 132 - 8 | VAN DEN HOUT ET AL., LANCET, vol. 56, 2000, pages 397 - 8 | VAN DEN HOUT ET AL., PEDIATRICS, vol. 113, 2004, pages 448 - 57 | WINLCEL ET AL., ANN NEUROL., vol. 55, 2004, pages 495 - 502 | SUN ET AL., MOL THER., vol. 11, no. 1, 2005, pages 57 - 65 | GOLDSPIEL ET AL., CLINICAL PHARMACY, vol. 12, 1993, pages 488 - 505 | WU; WU, BIOTHERAPY, vol. 3, 1991, pages 87 - 95 | TOLSTOSHEV, ANN. REV. PHARMACOL. TOXICOL., vol. 32, 1993, pages 573 - 596 | MULLIGAN, SCIENCE, vol. 260, 1993, pages 926 - 932 | MORGAN; ANDERSON, ANN. REV. BIOCHEM., vol. 62, 1993, pages 191 - 217 | MAY, TIBTECH, vol. 11, 1993, pages 155 - 215 | AUSUBEL ET AL.,: "Current Protocols in Molecular Biology", 1993, JOHN WILEY & SONS | KRIEGLER: "Gene Transfer and Expression, A Laboratory Manual", 1990, STOCKTON PRESS | DRACOPOLI ET AL.,: "Current Protocols in Human Genetics", 1994, JOHN WILEY & SONS, article "Chapters 12 and 13," | COLOSIMO ET AL., BIOTECHNIQUES, vol. 29, no. 2, 2000, pages 314 - 8,320-2,324 | SAMBROOK; FRITSCH; MANIATIS: "Molecular Cloning: A Laboratory Manual, Second Edition", 1989, COLD SPRING HARBOR LABORATORY PRESS | D.N. GLOVER: "DNA Cloning: A Practical Approach", vol. I, II, 1985 | M.J. GAIT: "Oligonucleotide Synthesis", 1984 | B.D. HAMES & S.J.EHIGGINS: "Nucleic Acid Hybridization", 1985 | B.D. HAMES & S.J. HIGGINS: "Transcription And Translation", 1984 | R.I. FRESHNEY,: "Animal Cell Culture", 1986 | "Immobilized Cells And Enzymes", 1986, IRL PRESS | B.E PERBAL: "A Practical Guide To Molecular Cloning", 1984 | MCCONNELL ET AL., HUM GENE THER., vol. 15, no. 11, 2004, pages 1022 - 33 | MCCARTY ET AL., ANNU REV GENET., vol. 38, 2004, pages 819 - 45 | MAH ET AL., CLIN. PHARMACOKINET, vol. 41, no. 12, 2002, pages 901 - 11 | SCOTT ET AL., NEUROMUSCU . DISORD., vol. 12, 2002, pages 23 - 9 | BECK ET AL., CURR GENE THER., vol. 4, no. 4, 2004, pages 457 - 67 | KOLLER; SMITHIES, PROC. NATL. ACAD. SCI. USA., vol. 86, 1989, pages 8932 - 8935 | ZIJLSTRA ET AL., NATURE, vol. 342, 1989, pages 435 - 438 | J BIOL. CHEM., vol. 62, 1987, pages 4429 - 4432 | MI ET AL., MOL. THERAPY., vol. 2, 2000, pages 339 - 47 | SCHERER ET AL., GENE THERAPY, vol. 9, 2002, pages 102 - 9 | FELGNER; RINGOLD, NATURE, vol. 337, 1989, pages 387 | BRIGHAM, AM. J MED. SCI., vol. 298, 1989, pages 278 | OSAKA ET AL., J PHARM. SCI., vol. 85, no. 6, 1996, pages 612 - 618 | SAN ET AL., HUMAN GENE THERAPY, vol. 4, 1993, pages 781 - 788 | SENIOR ET AL., BIOCHEMICA ET BIOPHYSICA ACTA., vol. 1070, 1991, pages 173 - 179 | KABANOV; KABANOV, BIOCONJUGATE CHEM., vol. 6, 1995, pages 7 - 20 | LIU ET AL., PHARMACEUT. RES., 1996, pages 13 | REMY ET AL., BIOCONJUGATE CHEN ., vol. 5, 1994, pages 647 - 654 | BEHR, J-P., BIOCONJUGATE CHEM., vol. 5, 1994, pages 382 - 389 | WYMAN ET AL., BIOCHEM., vol. 36, 1997, pages 3008 - 3017 | PFUTZNER ET AL., EXPERT OPIN. INVESTIG. DRUGS., vol. 9, 2000, pages 2069 - 83 | BOSHAGEN; GEIGER; JUNGE, ANGEWANTE CHEMIE, INT. ED. ENGL., vol. 20, no. 9, 1981, pages 806 - 807 | HALLIOGLO ET AL., PEDIATR INT., vol. 43, no. 4, 2001, pages 400 - 4 | PIPER ET AL.: "Motor Assessment of the Developing Infant", 1994, W.B. SAUNDERS CO. | BAYLEY ET AL.: "Bayley Scores of Infant Development. 2nd Ed", 1993, HARCOURT BRACE & CO. |