AU2010233188B2 - Methods for preventing and/or treating lysosomal storage disorders - Google Patents
Methods for preventing and/or treating lysosomal storage disorders Download PDFInfo
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- AU2010233188B2 AU2010233188B2 AU2010233188A AU2010233188A AU2010233188B2 AU 2010233188 B2 AU2010233188 B2 AU 2010233188B2 AU 2010233188 A AU2010233188 A AU 2010233188A AU 2010233188 A AU2010233188 A AU 2010233188A AU 2010233188 B2 AU2010233188 B2 AU 2010233188B2
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- Prior art keywords
- diol
- piperdine
- compound
- chloromethyl
- disease
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- BDERNNFJNOPAEC-UHFFFAOYSA-N propan-1-ol Chemical compound CCCO BDERNNFJNOPAEC-UHFFFAOYSA-N 0.000 description 1
- 235000010232 propyl p-hydroxybenzoate Nutrition 0.000 description 1
- QELSKZZBTMNZEB-UHFFFAOYSA-N propylparaben Chemical class CCCOC(=O)C1=CC=C(O)C=C1 QELSKZZBTMNZEB-UHFFFAOYSA-N 0.000 description 1
- UMJSCPRVCHMLSP-UHFFFAOYSA-N pyridine Natural products COC1=CC=CN=C1 UMJSCPRVCHMLSP-UHFFFAOYSA-N 0.000 description 1
- WKSAUQYGYAYLPV-UHFFFAOYSA-N pyrimethamine Chemical compound CCC1=NC(N)=NC(N)=C1C1=CC=C(Cl)C=C1 WKSAUQYGYAYLPV-UHFFFAOYSA-N 0.000 description 1
- 229960000611 pyrimethamine Drugs 0.000 description 1
- 229940073095 questran Drugs 0.000 description 1
- 230000001105 regulatory effect Effects 0.000 description 1
- 239000003201 reproductive control agent Substances 0.000 description 1
- 229920005989 resin Polymers 0.000 description 1
- 239000011347 resin Substances 0.000 description 1
- 239000000407 respiratory system agent Substances 0.000 description 1
- 150000003873 salicylate salts Chemical class 0.000 description 1
- 239000000523 sample Substances 0.000 description 1
- 210000003786 sclera Anatomy 0.000 description 1
- 238000012163 sequencing technique Methods 0.000 description 1
- 208000011985 sialidosis Diseases 0.000 description 1
- DQTKLICLJUKNCG-ZTYPAOSTSA-N siastatin b Chemical compound CC(=O)N[C@H]1NC[C@H](C(O)=O)[C@H](O)[C@@H]1O DQTKLICLJUKNCG-ZTYPAOSTSA-N 0.000 description 1
- 239000000377 silicon dioxide Substances 0.000 description 1
- 150000003384 small molecules Chemical class 0.000 description 1
- 239000001509 sodium citrate Substances 0.000 description 1
- NLJMYIDDQXHKNR-UHFFFAOYSA-K sodium citrate Chemical compound O.O.[Na+].[Na+].[Na+].[O-]C(=O)CC(O)(CC([O-])=O)C([O-])=O NLJMYIDDQXHKNR-UHFFFAOYSA-K 0.000 description 1
- 235000019333 sodium laurylsulphate Nutrition 0.000 description 1
- 239000012064 sodium phosphate buffer Substances 0.000 description 1
- 229940079832 sodium starch glycolate Drugs 0.000 description 1
- 239000008109 sodium starch glycolate Substances 0.000 description 1
- 229920003109 sodium starch glycolate Polymers 0.000 description 1
- 238000001228 spectrum Methods 0.000 description 1
- 238000013222 sprague-dawley male rat Methods 0.000 description 1
- 230000001954 sterilising effect Effects 0.000 description 1
- 238000004659 sterilization and disinfection Methods 0.000 description 1
- 239000007929 subcutaneous injection Substances 0.000 description 1
- 238000010254 subcutaneous injection Methods 0.000 description 1
- 239000000126 substance Substances 0.000 description 1
- 150000003890 succinate salts Chemical class 0.000 description 1
- 150000003467 sulfuric acid derivatives Chemical class 0.000 description 1
- 239000004094 surface-active agent Substances 0.000 description 1
- 239000000375 suspending agent Substances 0.000 description 1
- 239000000725 suspension Substances 0.000 description 1
- 238000013268 sustained release Methods 0.000 description 1
- 239000012730 sustained-release form Substances 0.000 description 1
- FXUAIOOAOAVCGD-FKSUSPILSA-N swainsonine Chemical compound C1CC[C@H](O)[C@H]2[C@H](O)[C@H](O)CN21 FXUAIOOAOAVCGD-FKSUSPILSA-N 0.000 description 1
- FXUAIOOAOAVCGD-UHFFFAOYSA-N swainsonine Natural products C1CCC(O)C2C(O)C(O)CN21 FXUAIOOAOAVCGD-UHFFFAOYSA-N 0.000 description 1
- 229960005566 swainsonine Drugs 0.000 description 1
- 239000003765 sweetening agent Substances 0.000 description 1
- 230000008961 swelling Effects 0.000 description 1
- 239000006188 syrup Substances 0.000 description 1
- 235000020357 syrup Nutrition 0.000 description 1
- 230000009885 systemic effect Effects 0.000 description 1
- 239000000454 talc Substances 0.000 description 1
- 229910052623 talc Inorganic materials 0.000 description 1
- 238000004885 tandem mass spectrometry Methods 0.000 description 1
- 150000003892 tartrate salts Chemical class 0.000 description 1
- 235000019157 thiamine Nutrition 0.000 description 1
- KYMBYSLLVAOCFI-UHFFFAOYSA-N thiamine Chemical compound CC1=C(CCO)SCN1CC1=CN=C(C)N=C1N KYMBYSLLVAOCFI-UHFFFAOYSA-N 0.000 description 1
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- 239000011721 thiamine Substances 0.000 description 1
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- JOXIMZWYDAKGHI-UHFFFAOYSA-N toluene-4-sulfonic acid Chemical compound CC1=CC=C(S(O)(=O)=O)C=C1 JOXIMZWYDAKGHI-UHFFFAOYSA-N 0.000 description 1
- FGMPLJWBKKVCDB-UHFFFAOYSA-N trans-L-hydroxy-proline Natural products ON1CCCC1C(O)=O FGMPLJWBKKVCDB-UHFFFAOYSA-N 0.000 description 1
- 125000005270 trialkylamine group Chemical group 0.000 description 1
- OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Natural products OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 description 1
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Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
- C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
- C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
- C07D211/36—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/90—Enzymes; Proenzymes
- G01N2333/914—Hydrolases (3)
- G01N2333/924—Hydrolases (3) acting on glycosyl compounds (3.2)
Landscapes
- Chemical & Material Sciences (AREA)
- Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Microbiology (AREA)
- Immunology (AREA)
- Genetics & Genomics (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Analytical Chemistry (AREA)
- Biophysics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Hospice & Palliative Care (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Psychiatry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Hydrogenated Pyridines (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US16810909P | 2009-04-09 | 2009-04-09 | |
| US61/168,109 | 2009-04-09 | ||
| PCT/US2010/030472 WO2010118283A1 (en) | 2009-04-09 | 2010-04-09 | Methods for preventing and/or treating lysosomal storage disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2010233188A1 AU2010233188A1 (en) | 2011-11-03 |
| AU2010233188B2 true AU2010233188B2 (en) | 2015-07-16 |
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|---|---|---|---|
| AU2010233188A Ceased AU2010233188B2 (en) | 2009-04-09 | 2010-04-09 | Methods for preventing and/or treating lysosomal storage disorders |
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| Country | Link |
|---|---|
| US (1) | US8940766B2 (enExample) |
| EP (1) | EP2416656B1 (enExample) |
| JP (1) | JP5645918B2 (enExample) |
| CN (1) | CN102625661B (enExample) |
| AU (1) | AU2010233188B2 (enExample) |
| BR (1) | BRPI1010517A2 (enExample) |
| CA (1) | CA2758271C (enExample) |
| ES (1) | ES2564093T3 (enExample) |
| IL (1) | IL215583A (enExample) |
| NZ (1) | NZ595629A (enExample) |
| WO (1) | WO2010118283A1 (enExample) |
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| JP2008545657A (ja) | 2005-05-17 | 2008-12-18 | アミカス セラピューティックス インコーポレイテッド | １−デオキシノジリマイシンおよび誘導体を用いるポンペ病の治療方法 |
| ES2505243T3 (es) * | 2009-04-09 | 2014-10-09 | Amicus Therapeutics, Inc. | Agentes terapéuticos para prevenir y/o tratar trastornos degenerativos del sistema nervioso central |
| LT2995306T (lt) | 2009-10-19 | 2019-04-10 | Amicus Therapeutics, Inc. | Naujos kompozicijos lizosomų kaupimosi sutrikimų profilaktikai ir (arba) gydymui |
| DK2490532T3 (en) | 2009-10-19 | 2017-02-27 | Amicus Therapeutics Inc | Hitherto UNKNOWN COMPOSITIONS FOR PREVENTION AND / OR TREATMENT OF DEGENERATIVE DISORDERS IN THE CENTRAL Nervous System |
| EP2823043A4 (en) | 2012-03-07 | 2016-09-07 | Amicus Therapeutics Inc | HIGHLY CONCENTRATED ALPHA-GLUCOSIDASE COMPOSITIONS FOR THE TREATMENT OF MORBUS POMPE |
| JP2017505350A (ja) * | 2014-02-12 | 2017-02-16 | アミカス セラピューティックス インコーポレイテッド | 硫黄含有部分を含む糖誘導体ならびにその作製方法およびｍｐｓ ｉｉｉｃの処置のためのその使用方法 |
| BR112017005810A2 (pt) | 2014-09-30 | 2018-02-27 | Amicus Therapeutics Inc | alfa-glucosidase ácida altamente potente com carboidratos melhorados |
| IL299470A (en) | 2015-12-30 | 2023-02-01 | Amicus Therapeutics Inc | Improved acid alpha-glucosidase for the treatment of Pompe disease |
| PE20190127A1 (es) | 2016-03-30 | 2019-01-17 | Amicus Therapeutics Inc | Metodo para seleccionar proteinas recombinantes ricas en m6p |
| AU2017239641A1 (en) | 2016-03-30 | 2018-10-18 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
| LT3624831T (lt) | 2017-05-15 | 2023-07-10 | Amicus Therapeutics, Inc. | Rekombinantinė žmogaus rūgštinė alfa-glukozidazė |
| US20220184185A1 (en) | 2018-07-25 | 2022-06-16 | Modernatx, Inc. | Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20050130972A1 (en) * | 2003-11-12 | 2005-06-16 | Amicus Therapeutics, Inc. | Hydroxy piperidine derivatives to treat gaucher disease |
| US20080009516A1 (en) * | 2006-06-23 | 2008-01-10 | Wustman Brandon A | Method for the treatment of neurological disorders by enhancing the activity of beta-glucocerebrosidase |
| WO2008134628A2 (en) * | 2007-04-26 | 2008-11-06 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
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|---|---|---|---|---|
| US4407957A (en) | 1981-03-13 | 1983-10-04 | Damon Corporation | Reversible microencapsulation of a core material |
| ES2107537T3 (es) | 1991-04-25 | 1997-12-01 | Univ Brown Res Found | Vehiculo inmunoaislante biocompatible implantable para suministrar productos terapeuticos seleccionados. |
| US5383851A (en) | 1992-07-24 | 1995-01-24 | Bioject Inc. | Needleless hypodermic injection device |
| US5569189A (en) | 1992-09-28 | 1996-10-29 | Equidyne Systems, Inc. | hypodermic jet injector |
| AU688283B2 (en) | 1992-09-29 | 1998-03-12 | Novartis Ag | Pulmonary delivery of active fragments of parathyroid hormone |
| AU1890295A (en) | 1994-03-09 | 1995-09-25 | Novo Nordisk A/S | Piperidines and pyrrolidines |
| FR2718357B1 (fr) | 1994-04-06 | 1997-10-03 | Defarges Alain Moreau | Perfectionnements apportés à un dispositif d'injection par jet sans aiguille. |
| US5844102A (en) | 1994-07-07 | 1998-12-01 | University Of Maryland Baltimore County | Glycohydrolase inhibitors, their preparation and use thereof |
| US5599302A (en) | 1995-01-09 | 1997-02-04 | Medi-Ject Corporation | Medical injection system and method, gas spring thereof and launching device using gas spring |
| US5780014A (en) | 1995-04-14 | 1998-07-14 | Inhale Therapeutic Systems | Method and apparatus for pulmonary administration of dry powder alpha 1-antitrypsin |
| US5654007A (en) | 1995-06-07 | 1997-08-05 | Inhale Therapeutic Systems | Methods and system for processing dispersible fine powders |
| US20040204379A1 (en) * | 2000-06-19 | 2004-10-14 | Cheng Seng H. | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| AU2002306848A1 (en) * | 2001-03-23 | 2002-10-08 | Elan Pharmaceuticals, Inc. | Methods of treating alzheimer's disease with piperidin derivates |
| JP2006507359A (ja) * | 2002-10-28 | 2006-03-02 | ノボ・ノルデイスク・エー／エス | 心循環器疾患の治療のためのグリコーゲンホスホリラーゼ阻害剤の使用 |
| US7446098B2 (en) | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
| US8321148B2 (en) | 2008-10-24 | 2012-11-27 | Amicus Therapeutics, Inc. | Multiple compartment dosing model |
| ES2505243T3 (es) * | 2009-04-09 | 2014-10-09 | Amicus Therapeutics, Inc. | Agentes terapéuticos para prevenir y/o tratar trastornos degenerativos del sistema nervioso central |
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2010
- 2010-04-09 ES ES10762469.4T patent/ES2564093T3/es active Active
- 2010-04-09 NZ NZ595629A patent/NZ595629A/xx not_active IP Right Cessation
- 2010-04-09 JP JP2012504881A patent/JP5645918B2/ja not_active Expired - Fee Related
- 2010-04-09 US US12/757,160 patent/US8940766B2/en active Active
- 2010-04-09 BR BRPI1010517A patent/BRPI1010517A2/pt not_active Application Discontinuation
- 2010-04-09 CN CN201080023544.XA patent/CN102625661B/zh not_active Expired - Fee Related
- 2010-04-09 WO PCT/US2010/030472 patent/WO2010118283A1/en not_active Ceased
- 2010-04-09 EP EP10762469.4A patent/EP2416656B1/en not_active Not-in-force
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- 2010-04-09 AU AU2010233188A patent/AU2010233188B2/en not_active Ceased
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Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20050130972A1 (en) * | 2003-11-12 | 2005-06-16 | Amicus Therapeutics, Inc. | Hydroxy piperidine derivatives to treat gaucher disease |
| US20080009516A1 (en) * | 2006-06-23 | 2008-01-10 | Wustman Brandon A | Method for the treatment of neurological disorders by enhancing the activity of beta-glucocerebrosidase |
| WO2008134628A2 (en) * | 2007-04-26 | 2008-11-06 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
Also Published As
| Publication number | Publication date |
|---|---|
| CN102625661A (zh) | 2012-08-01 |
| EP2416656B1 (en) | 2015-12-23 |
| JP5645918B2 (ja) | 2014-12-24 |
| US20100260740A1 (en) | 2010-10-14 |
| ES2564093T3 (es) | 2016-03-17 |
| NZ595629A (en) | 2013-04-26 |
| US8940766B2 (en) | 2015-01-27 |
| WO2010118283A1 (en) | 2010-10-14 |
| IL215583A0 (en) | 2011-12-29 |
| CA2758271A1 (en) | 2010-10-14 |
| BRPI1010517A2 (pt) | 2017-01-31 |
| CN102625661B (zh) | 2015-05-27 |
| CA2758271C (en) | 2018-06-05 |
| EP2416656A1 (en) | 2012-02-15 |
| AU2010233188A1 (en) | 2011-11-03 |
| JP2012523431A (ja) | 2012-10-04 |
| EP2416656A4 (en) | 2012-09-05 |
| IL215583A (en) | 2016-09-29 |
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| AU2010233188B2 (en) | Methods for preventing and/or treating lysosomal storage disorders | |
| EP2995306B1 (en) | Novel compositions for preventing and/or treating lysosomal storage disorders | |
| AU2010233187B2 (en) | Methods for preventing and/or treating degenerative disorders of the central nervous system | |
| HK1174201B (en) | Novel compositions for preventing and/or treating lysosomal storage disorders |
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| FGA | Letters patent sealed or granted (standard patent) | ||
| MK14 | Patent ceased section 143(a) (annual fees not paid) or expired |