PL2402002T3 - Kompozycje zawierające kwas 1,2,4-oksadiazolobenzoesowy i ich zastosowania do leczenia chorób związanych z przedwczesnym kodonem stop - Google Patents

Kompozycje zawierające kwas 1,2,4-oksadiazolobenzoesowy i ich zastosowania do leczenia chorób związanych z przedwczesnym kodonem stop

Info

Publication number
PL2402002T3
PL2402002T3 PL11175569T PL11175569T PL2402002T3 PL 2402002 T3 PL2402002 T3 PL 2402002T3 PL 11175569 T PL11175569 T PL 11175569T PL 11175569 T PL11175569 T PL 11175569T PL 2402002 T3 PL2402002 T3 PL 2402002T3
Authority
PL
Poland
Prior art keywords
oxadiazole
compositions
stop codon
diseases associated
treating diseases
Prior art date
Application number
PL11175569T
Other languages
English (en)
Inventor
Samit Hirawat
Langdon Miller
Original Assignee
Ptc Therapeutics, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ptc Therapeutics, Inc. filed Critical Ptc Therapeutics, Inc.
Publication of PL2402002T3 publication Critical patent/PL2402002T3/pl

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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
    • A61K9/145—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic compounds
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PL11175569T 2005-04-08 2006-04-06 Kompozycje zawierające kwas 1,2,4-oksadiazolobenzoesowy i ich zastosowania do leczenia chorób związanych z przedwczesnym kodonem stop PL2402002T3 (pl)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US66978905P 2005-04-08 2005-04-08
US78739506P 2006-03-30 2006-03-30
EP11175569.0A EP2402002B1 (en) 2005-04-08 2006-04-06 Compositions comprising an 1,2,4-oxadiazole and uses thereof for treating diseases associated with a premature stop codon
PCT/US2006/012887 WO2006110483A1 (en) 2005-04-08 2006-04-06 Compositions of an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy
EP06749439A EP1874306B1 (en) 2005-04-08 2006-04-06 Compositions of an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy

Publications (1)

Publication Number Publication Date
PL2402002T3 true PL2402002T3 (pl) 2018-11-30

Family

ID=36763092

Family Applications (2)

Application Number Title Priority Date Filing Date
PL11175569T PL2402002T3 (pl) 2005-04-08 2006-04-06 Kompozycje zawierające kwas 1,2,4-oksadiazolobenzoesowy i ich zastosowania do leczenia chorób związanych z przedwczesnym kodonem stop
PL06749439T PL1874306T3 (pl) 2005-04-08 2006-04-06 Kompozycje zawierające 1,2,4-oksadiazol aktywny po podaniu doustnie w celu supresyjnego leczenia niesensownej mutacji

Family Applications After (1)

Application Number Title Priority Date Filing Date
PL06749439T PL1874306T3 (pl) 2005-04-08 2006-04-06 Kompozycje zawierające 1,2,4-oksadiazol aktywny po podaniu doustnie w celu supresyjnego leczenia niesensownej mutacji

Country Status (17)

Country Link
US (7) US8716321B2 (pl)
EP (2) EP2402002B1 (pl)
JP (3) JP5800451B2 (pl)
CN (1) CN103720688A (pl)
AU (1) AU2006235115C1 (pl)
BR (1) BRPI0609089A2 (pl)
CA (1) CA2603402C (pl)
DK (2) DK2402002T3 (pl)
ES (2) ES2390804T3 (pl)
HK (1) HK1111897A1 (pl)
IL (2) IL186433A (pl)
MX (1) MX2007012206A (pl)
NO (2) NO339909B1 (pl)
NZ (1) NZ562197A (pl)
PL (2) PL2402002T3 (pl)
PT (2) PT2402002T (pl)
WO (1) WO2006110483A1 (pl)

Families Citing this family (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3345895T3 (pl) 2003-04-11 2020-05-18 Ptc Therapeutics, Inc. Związek kwasu 1,2,4-oksadiazolobenzoesowego i jego zastosowanie do supresji mutacji nonsensownych i leczenia choroby
PL2402002T3 (pl) 2005-04-08 2018-11-30 Ptc Therapeutics, Inc. Kompozycje zawierające kwas 1,2,4-oksadiazolobenzoesowy i ich zastosowania do leczenia chorób związanych z przedwczesnym kodonem stop
BRPI0710213A2 (pt) 2006-03-30 2011-04-12 Ptc Therapeutics Inc métodos para produzir uma quantidade efetiva de uma proteìna de transleitura funcional codificada por uma sequência de ácido nucleico que compreende uma mutação não-sentido e para tratar, controlar e/ou prevenir uma doença
US7645789B2 (en) 2006-04-07 2010-01-12 Vertex Pharmaceuticals Incorporated Indole derivatives as CFTR modulators
US10022352B2 (en) 2006-04-07 2018-07-17 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
ES2882684T3 (es) 2006-04-07 2021-12-02 Vertex Pharma Preparación de moduladores de transportadores del casete de unión a ATP
ZA200901782B (en) 2006-09-08 2010-06-30 Ptc Therapeutics Inc Processes for the preparation of 1, 2, 4-oxadiazole benzoic acids
US8101641B2 (en) 2006-09-25 2012-01-24 Ptc Therapeutics, Inc. Hydroxylated 1,2,4-oxadiazole benzoic acid compounds and compositions thereof
SG185979A1 (en) * 2006-09-25 2012-12-28 Ptc Therapeutics Inc 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
AU2016262645B2 (en) * 2006-10-12 2018-05-17 Ptc Therapeutics, Inc. Methods for dosing an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy
US8563573B2 (en) 2007-11-02 2013-10-22 Vertex Pharmaceuticals Incorporated Azaindole derivatives as CFTR modulators
MX2009012558A (es) 2007-08-03 2010-04-21 Biomarin Iga Ltd Combinaciones de farmacos para el tratamiento de distrofia muscular de duchenne.
GB0715087D0 (en) 2007-08-03 2007-09-12 Summit Corp Plc Drug combinations for the treatment of duchenne muscular dystrophy
NZ598941A (en) 2007-11-16 2013-06-28 Vertex Pharma Isoquinoline modulators of ATP-Binding Cassette transporters
DK3170818T3 (da) 2007-12-07 2020-04-14 Vertex Pharma Faste former af 3-(6-(1-(2,2-difluorbenzo[d][1,3]dioxol-5-yl) cyclopropancarboxamido)-3-methylpyridin-2-yl)benzoesyre
EP2980077B1 (en) 2008-03-31 2020-05-06 Vertex Pharmaceuticals Incorporated Pyridyl derivatives as cftr modulators
EP4253381A3 (en) 2010-03-25 2023-11-01 Vertex Pharmaceuticals Incorporated Solid forms of (r)-1-(2,2-difluorobenzo[d][1,3]d ioxol-5-yl)-n-(1-(2,3-dihydroxyp ropyl)-6-fluoro-2-(1-hydroxy- 2-methylpropan-2-yl)-1h-indol- 5-yl) cyclopropanecarboxamide
US8802868B2 (en) 2010-03-25 2014-08-12 Vertex Pharmaceuticals Incorporated Solid forms of (R)-1(2,2-difluorobenzo[D][1,3]dioxo1-5-yl)-N-(1-(2,3-dihydroxypropyl-6-fluoro-2-(1-hydroxy-2-methylpropan2-yl)-1H-Indol-5-yl)-Cyclopropanecarboxamide
RS54783B1 (sr) 2010-04-07 2016-10-31 Vertex Pharma Čvrste forme 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioksol-5-il)ciklopropankarboksiamido)-3-metilpiridin-2-il)benzoeve kiseline
DK3150198T3 (da) 2010-04-07 2021-11-01 Vertex Pharma Farmaceutiske sammensætninger af 3-(6-(1-(2,2-difluorbenzo[d][1,3]dioxol-5-yl)-cyclopropancarboxamido)-3-methylpyriodin-2-yl)benzoesyre og indgivelse deraf
CN105130948A (zh) 2010-04-22 2015-12-09 弗特克斯药品有限公司 制备环烷基甲酰胺基-吲哚化合物的方法
US8563593B2 (en) 2010-06-08 2013-10-22 Vertex Pharmaceuticals Incorporated Formulations of (R)-1-(2,2-difluorobenzo[D] [1,3] dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl)cyclopropanecarboxamide
CN103189060A (zh) * 2010-08-05 2013-07-03 里尔法律与医疗卫生第二大学 用于对无义突变介导的疾病进行治疗的化合物和含有所述化合物的药物组合物
CA2808501A1 (en) 2010-08-23 2012-03-01 Vertex Pharmaceuticals Incorporated Pharmaceutical composition of (r)-1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)-n-(1-(2,3-dihydroxy propyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1h-indol-5-yl) cyclopropanecarboxamide and administration therof
US9254291B2 (en) 2011-11-08 2016-02-09 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
ES2742277T3 (es) 2012-01-25 2020-02-13 Vertex Pharma Composiciones farmacéuticas de ácido 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-ilo) ciclopropano carboxamido)-3metilpiridin-2-ilo)benzoico y su administración
CA2878057A1 (en) 2012-07-16 2014-01-23 Rossitza Gueorguieva Alargova Pharmaceutical compositions of (r)-1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)-n-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1h-indol-5-yl) cyclopropanecarboxamide and administration thereof
LT2914248T (lt) 2012-11-02 2018-11-26 Vertex Pharmaceuticals Incorporated Farmacinės kompozicijos, skirtos cftr medijuotų ligų gydymui
WO2014153643A1 (en) * 2013-03-26 2014-10-02 The University Of British Columbia Compositions and methods for use thereof in the treatment of aniridia
CA2942147C (en) * 2014-03-06 2022-12-13 Ptc Therapeutics, Inc. Pharmaceutical compositions and salts of a 1,2,4-oxadiazole benzoic acid
RS57476B9 (sr) 2014-04-15 2021-10-29 Vertex Pharma Farmaceutske kompozicije za lečenje bolesti posredovanih transmembranskim regulatorom provodljivosti cistične fibroze
US20170196842A1 (en) * 2014-06-06 2017-07-13 Ptc Therapeutics, Inc. Methods of treatment using a 1,2,4-oxadiazole benzoic acid
WO2016057572A1 (en) 2014-10-06 2016-04-14 Mark Thomas Miller Modulators of cystic fibrosis transmembrane conductance regulator
WO2017075312A1 (en) 2015-10-30 2017-05-04 Ptc Therapeutics, Inc. Methods for treating epilepsy
CA3048204A1 (en) 2015-12-23 2017-06-29 Moonshot Pharma Llc Methods for inducing an immune response
TW201808922A (zh) * 2016-06-20 2018-03-16 台灣神隆股份有限公司 製備阿塔魯仁及其中間體的方法
WO2018050706A1 (en) * 2016-09-13 2018-03-22 Marco Cipolli Method of treatment of shwachman-diamond syndrome
WO2018237326A1 (en) 2017-06-22 2018-12-27 1AlMOONSHOT PHARMA LLC METHODS OF TREATING CANCER USING COMPOSITIONS COMPRISING AMLEXANOX AND IMMUNOMODULATORS
ES2964964T3 (es) 2017-11-01 2024-04-10 Bristol Myers Squibb Co Compuestos bicíclicos puenteados como moduladores del receptor X farnesoide
WO2019101709A1 (en) 2017-11-23 2019-05-31 Universita' Degli Studi Di Palermo Oxadiazole derivatives for the treatment of genetic diseases due to nonsense mutations
WO2020128816A2 (en) 2018-12-20 2020-06-25 Pfizer Inc. Pharmaceutical compositions and methods comprising a combination of a benzoxazole transthyretin stabilizer and an additional therapeutic agent
AR118050A1 (es) 2019-02-15 2021-09-15 Bristol Myers Squibb Co Compuestos bicíclicos sustituidos como moduladores del receptor farnesoide x
JP2022519771A (ja) 2019-02-15 2022-03-24 ブリストル－マイヤーズ スクイブ カンパニー ファルネソイドｘ受容体モジュレータとして有用な置換アミド化合物
JP7449300B2 (ja) 2019-02-15 2024-03-13 ブリストル－マイヤーズ スクイブ カンパニー ファルネソイドｘ受容体モジュレータとして有用な置換アミド化合物
AU2020221370A1 (en) 2019-02-15 2021-10-07 Bristol-Myers Squibb Company Substituted bicyclic compounds as farnesoid x receptor modulators
WO2022112919A1 (en) 2020-11-25 2022-06-02 Pfizer Inc. (aza)benzothiazolyl substituted pyrazole compounds
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

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MY128323A (en) * 1996-09-30 2007-01-31 Otsuka Pharma Co Ltd Thiazole derivatives for inhibition of cytokine production and of cell adhesion
FI107732B (fi) * 1998-03-18 2001-09-28 Xyrofin Oy Laktitolin kiteyttäminen, kiteinen laktitolituote ja sen käyttö
US6660753B2 (en) * 1999-08-19 2003-12-09 Nps Pharmaceuticals, Inc. Heteropolycyclic compounds and their use as metabotropic glutamate receptor antagonists
US6316029B1 (en) 2000-05-18 2001-11-13 Flak Pharma International, Ltd. Rapidly disintegrating solid oral dosage form
EP1406632A4 (en) * 2001-06-08 2009-11-04 Cytovia Inc 3-ARYL-5-ARYL-1,2,4-OXADIAZOLES AND THEIR ANALOGUES, CASPASE ACTIVATORS AND INDUCERS OF APOPTOSIS, AND USES THEREOF
MY151199A (en) * 2001-11-02 2014-04-30 Rigel Pharmaceuticals Inc Substituted diphenyl heterocycles useful for treating hcv infection
TW200812986A (en) * 2002-08-09 2008-03-16 Nps Pharma Inc New compounds
US20040253307A1 (en) * 2003-02-04 2004-12-16 Brian Hague Sugar-free oral transmucosal solid dosage forms and uses thereof
GB0303503D0 (en) * 2003-02-14 2003-03-19 Novartis Ag Organic compounds
PL3345895T3 (pl) * 2003-04-11 2020-05-18 Ptc Therapeutics, Inc. Związek kwasu 1,2,4-oksadiazolobenzoesowego i jego zastosowanie do supresji mutacji nonsensownych i leczenia choroby
US20050075375A1 (en) * 2003-05-14 2005-04-07 Anadys Pharmaceuticals, Inc. Heterocyclic compounds for treating hepatitis C virus
PL2402002T3 (pl) 2005-04-08 2018-11-30 Ptc Therapeutics, Inc. Kompozycje zawierające kwas 1,2,4-oksadiazolobenzoesowy i ich zastosowania do leczenia chorób związanych z przedwczesnym kodonem stop
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
SG185979A1 (en) * 2006-09-25 2012-12-28 Ptc Therapeutics Inc Crystalline forms of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid
CA2942147C (en) 2014-03-06 2022-12-13 Ptc Therapeutics, Inc. Pharmaceutical compositions and salts of a 1,2,4-oxadiazole benzoic acid

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