HRP20150590T1 - Postupak za doziranje oralno aktivnog 1,2,4-oksadiazola u terapiji za suzbijanje non-sense mutacije - Google Patents
Postupak za doziranje oralno aktivnog 1,2,4-oksadiazola u terapiji za suzbijanje non-sense mutacije Download PDFInfo
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- HRP20150590T1 HRP20150590T1 HRP20150590TT HRP20150590T HRP20150590T1 HR P20150590 T1 HRP20150590 T1 HR P20150590T1 HR P20150590T T HRP20150590T T HR P20150590TT HR P20150590 T HRP20150590 T HR P20150590T HR P20150590 T1 HRP20150590 T1 HR P20150590T1
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- 238000000034 method Methods 0.000 title claims 3
- 108020004485 Nonsense Codon Proteins 0.000 title claims 2
- BBVIDBNAYOIXOE-UHFFFAOYSA-N 1,2,4-oxadiazole Chemical compound C=1N=CON=1 BBVIDBNAYOIXOE-UHFFFAOYSA-N 0.000 title 1
- 230000037434 nonsense mutation Effects 0.000 title 1
- 230000001629 suppression Effects 0.000 title 1
- 238000002560 therapeutic procedure Methods 0.000 title 1
- 150000001875 compounds Chemical class 0.000 claims 15
- OOUGLTULBSNHNF-UHFFFAOYSA-N 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid Chemical compound OC(=O)C1=CC=CC(C=2N=C(ON=2)C=2C(=CC=CC=2)F)=C1 OOUGLTULBSNHNF-UHFFFAOYSA-N 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 101150029409 CFTR gene Proteins 0.000 claims 1
- 201000003883 Cystic fibrosis Diseases 0.000 claims 1
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 1
- 108010069091 Dystrophin Proteins 0.000 claims 1
- 238000011340 continuous therapy Methods 0.000 claims 1
- 230000014509 gene expression Effects 0.000 claims 1
- 230000036470 plasma concentration Effects 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- 239000012453 solvate Substances 0.000 claims 1
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- 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
- A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
- A61K31/4245—Oxadiazoles
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
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- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
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- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/12—Carboxylic acids; Salts or anhydrides thereof
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- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
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- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K47/32—Macromolecular compounds obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. carbomers, poly(meth)acrylates, or polyvinyl pyrrolidone
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- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K47/34—Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
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- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
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- Animal Behavior & Ethology (AREA)
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- Engineering & Computer Science (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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- Diabetes (AREA)
- Neurology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
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- Hematology (AREA)
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- Orthopedic Medicine & Surgery (AREA)
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- Proteomics, Peptides & Aminoacids (AREA)
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Claims (10)
1. Spoj za upotrebu u postupku liječenja, sprječavanja ili ublažavanja bolesti povezane s nedostatkom ekspresije gena uslijed preuranjenog stop kodona, naznačen time, da je spoj 3-[5-(2-fluoro-fenil)-[1,2,4]oksadiazol-3-il]benzojeva kiselina, ili njegova farmaceutski prihvatljiva sol, solvat ili hidrat, pri čemu je navedena bolest cistična fibroza ili Duchenneova mišićna distrofija, i pri čemu rečeni postupak uključuje davanje spoja u tri dnevne doze, pri čemu se prva doza, druga doza i treća doza primjenjuje u razdoblju od 24 sata, prema formuli 1X, 1X, 2X, gdje je X 18-22 mg/kg, i pri čemu svaka doza održava razinu u plazmi 3-[5-(2-fluoro-fenil)-[1,2,4]oksadiazol-3-il]-benzojeve kiseline na više od 2 mg/ml u trajanju od najmanje 2 sata.
2. Spoj za primjenu prema zahtjevu 1, naznačen time, da se doze daju oralno ljudima.
3. Spoj za primjenu prema zahtjevu 1, naznačen time, da je gen CFTR gen.
4. Spoj za primjenu prema zahtjevu 1, naznačen time, da je gen distrofin gen.
5. Spoj za primjenu prema zahtjevu 1, naznačen time, da se spoj primjenjuje pomoću jedne ili više jedinica doze pripravka od kojih svaka sadrži količinu navedenog spoja u rasponu između 35 mg i 1400 mg.
6. Spoj za uporabu prema zahtjevu 5, naznačen time, da je količina spomenutog spoja u svakoj jedinici doze pripravka odabran između 35 mg, 50 mg, 70 mg, 100 mg, 125 mg, 140 mg, 175 mg, 200 mg, 250 mg , 280 mg, 350 mg, 500 mg, 560 mg, 700 mg, 750 mg, 1000 mg i 1400 mg.
7. Spoj za primjenu prema zahtjevu 1, naznačen time, da se prva doza primjenjuje ujutro, druga doza se primjenjuje popodne i treća doza se primjenjuje u večernjim satima razdoblja od 24 sata.
8. Spoj za primjenu prema zahtjevu 1, naznačen time, da se doze daju u intervalima od 4, 5, 6, 7, 8, 9, 10, 11, 12, 13 ili 14 sati između doza.
9. Spoj za uporabu prema zahtjevu 1, naznačen time, da se druga doza primjenjuje oko 6 sati nakon davanja prve doze, pri čemu se treća doza primjenjuje oko 6 sati nakon davanja doze i pri čemu se prva doza primjenjuje oko 12 sati nakon davanja treće doze u proteklom razdoblju od 24 sata, a prva doza, druga doza, odnosno treća doza primjenjuju se 30 minuta nakon jela.
10. Spoj za uporabu prema zahtjevu 1, naznačen time, da se tri dnevne doze primjenjuju u kontinuiranoj terapiji koja podrazumijeva više 24-satnih perioda.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US85145006P | 2006-10-12 | 2006-10-12 | |
| EP07852738.9A EP2073805B1 (en) | 2006-10-12 | 2007-10-11 | Methods for dosing an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy |
| PCT/US2007/021921 WO2008045566A1 (en) | 2006-10-12 | 2007-10-11 | Methods for dosing an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20150590T1 true HRP20150590T1 (hr) | 2015-07-03 |
Family
ID=39102963
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20150590TT HRP20150590T1 (hr) | 2006-10-12 | 2015-06-02 | Postupak za doziranje oralno aktivnog 1,2,4-oksadiazola u terapiji za suzbijanje non-sense mutacije |
Country Status (24)
| Country | Link |
|---|---|
| US (7) | US9226919B2 (hr) |
| EP (2) | EP2073805B1 (hr) |
| JP (4) | JP2010506840A (hr) |
| KR (4) | KR20090074231A (hr) |
| CN (2) | CN101553226B (hr) |
| AU (1) | AU2007308067B2 (hr) |
| BR (2) | BRPI0719276A2 (hr) |
| CA (1) | CA2675518C (hr) |
| CL (1) | CL2007002923A1 (hr) |
| DK (1) | DK2073805T3 (hr) |
| ES (1) | ES2538359T3 (hr) |
| HK (2) | HK1136214A1 (hr) |
| HR (1) | HRP20150590T1 (hr) |
| IL (2) | IL198147A (hr) |
| MX (1) | MX2009003909A (hr) |
| NO (1) | NO342061B1 (hr) |
| PE (4) | PE20080982A1 (hr) |
| PL (1) | PL2073805T3 (hr) |
| PT (1) | PT2073805E (hr) |
| RS (1) | RS54025B1 (hr) |
| RU (1) | RU2462247C2 (hr) |
| SI (1) | SI2073805T1 (hr) |
| TW (1) | TWI405570B (hr) |
| WO (1) | WO2008045566A1 (hr) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SI3345895T1 (sl) | 2003-04-11 | 2020-03-31 | Ptc Therapeutics, Inc. | Spojina 1,2,4-oksadiazol benzojske kisline in njena uporaba za supresijo nesmiselne in zdravljenje bolezni |
| EP2387995A1 (en) | 2006-03-30 | 2011-11-23 | PTC Therapeutics, Inc. | Methods for the production of functional protein from DNA having a nonsense mutation and the treatment of disorders associated therewith |
| RS54025B1 (en) | 2006-10-12 | 2015-10-30 | Ptc Therapeutics Inc. | DOSAGE PROCEDURES FOR ORALLY ACTIVE 1,2,4-OXADIAZOLE FOR NONSENS MUTATION SUPPRESSION THERAPY |
| EP2714937B1 (en) | 2011-06-03 | 2018-11-14 | Eisai R&D Management Co., Ltd. | Biomarkers for predicting and assessing responsiveness of thyroid and kidney cancer subjects to lenvatinib compounds |
| CA2942147C (en) | 2014-03-06 | 2022-12-13 | Ptc Therapeutics, Inc. | Pharmaceutical compositions and salts of a 1,2,4-oxadiazole benzoic acid |
| ES2926687T3 (es) | 2014-08-28 | 2022-10-27 | Eisai R&D Man Co Ltd | Derivado de quinolina muy puro y método para su producción |
| US20180028662A1 (en) | 2015-02-25 | 2018-02-01 | Eisai R&D Management Co., Ltd. | Method for Suppressing Bitterness of Quinoline Derivative |
| CA2988707C (en) | 2015-06-16 | 2023-10-10 | Eisai R&D Management Co., Ltd. | Combination of cbp/catenin inhibitor and immune checkpoint inhibitor for treating cancer |
| AR106530A1 (es) | 2015-10-30 | 2018-01-24 | Ptc Therapeutics Inc | Métodos para tratar epilepsia |
| JP7235741B2 (ja) | 2017-10-19 | 2023-03-08 | シプラ ユーエスエイ， インコーポレイテッド | 抗菌性アミノグリコシド類似体の合成 |
| EP3713934B1 (en) | 2017-11-23 | 2022-04-13 | Universita' degli Studi di Palermo | Oxadiazole derivatives for the treatment of genetic diseases due to nonsense mutations |
| US20240139183A1 (en) * | 2021-02-16 | 2024-05-02 | Ribotech Co., Ltd. | Compound for suppressing nonsense-mediated mrna decay |
| KR102378381B1 (ko) * | 2021-08-23 | 2022-03-24 | 라이보텍(주) | 넌센스-매개 mRNA 분해를 억제하기 위한 화합물 |
| EP4337267A1 (en) * | 2021-05-11 | 2024-03-20 | RegenxBio Inc. | Treatment of duchenne muscular dystrophy and combinations thereof |
| KR102665711B1 (ko) * | 2021-12-21 | 2024-05-14 | 재단법인 아산사회복지재단 | Ras 활성도를 이용한 신경섬유종증 치료제에 대한 감수성 예측용 바이오마커 조성물 및 Ras 활성 억제제를 포함하는 신경섬유종증 예방 또는 치료용 약학적 조성물 |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
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| MXPA05001592A (es) * | 2002-08-09 | 2005-05-05 | Astrazeneca Ab | Oxadiazoles como moduladores de receptor-5 de glutamato metabotropico. |
| GB0303503D0 (en) * | 2003-02-14 | 2003-03-19 | Novartis Ag | Organic compounds |
| SI3345895T1 (sl) * | 2003-04-11 | 2020-03-31 | Ptc Therapeutics, Inc. | Spojina 1,2,4-oksadiazol benzojske kisline in njena uporaba za supresijo nesmiselne in zdravljenje bolezni |
| WO2004110351A2 (en) * | 2003-05-14 | 2004-12-23 | Anadys Pharmaceuticals, Inc. | Heterocyclic compounds for treating hepatitis c virus |
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| ES2390804T3 (es) * | 2005-04-08 | 2012-11-16 | Ptc Therapeutics, Inc. | Composiciones de un 1,2,4-oxadiazol oralmente activo para terapia de supresión de la mutación sin sentido |
| US20070010553A1 (en) * | 2005-07-01 | 2007-01-11 | Astrazeneca Ab | New use |
| ZA200901782B (en) | 2006-09-08 | 2010-06-30 | Ptc Therapeutics Inc | Processes for the preparation of 1, 2, 4-oxadiazole benzoic acids |
| EP2559689B1 (en) | 2006-09-25 | 2017-11-29 | PTC Therapeutics, Inc. | Preparation process for crystalline forms of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazol-3-yl]benzoic acid |
| RS54025B1 (en) | 2006-10-12 | 2015-10-30 | Ptc Therapeutics Inc. | DOSAGE PROCEDURES FOR ORALLY ACTIVE 1,2,4-OXADIAZOLE FOR NONSENS MUTATION SUPPRESSION THERAPY |
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2007
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