PT2402002T - Composições de 1,2,4-oxadiazole oralmente activo para terapia de supressão de mutações nonsense - Google Patents

Composições de 1,2,4-oxadiazole oralmente activo para terapia de supressão de mutações nonsense

Info

Publication number
PT2402002T
PT2402002T PT11175569T PT11175569T PT2402002T PT 2402002 T PT2402002 T PT 2402002T PT 11175569 T PT11175569 T PT 11175569T PT 11175569 T PT11175569 T PT 11175569T PT 2402002 T PT2402002 T PT 2402002T
Authority
PT
Portugal
Prior art keywords
oxadiazole
compositions
stop codon
diseases associated
treating diseases
Prior art date
Application number
PT11175569T
Other languages
English (en)
Inventor
Miller Langdon
Hirawat Samit
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 PT2402002T publication Critical patent/PT2402002T/pt

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    • 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
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    • 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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PT11175569T 2005-04-08 2006-04-06 Composições de 1,2,4-oxadiazole oralmente activo para terapia de supressão de mutações nonsense PT2402002T (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US66978905P 2005-04-08 2005-04-08
US78739506P 2006-03-30 2006-03-30

Publications (1)

Publication Number Publication Date
PT2402002T true PT2402002T (pt) 2018-10-08

Family

ID=36763092

Family Applications (2)

Application Number Title Priority Date Filing Date
PT06749439T PT1874306E (pt) 2005-04-08 2006-04-06 Composições de um 1,2,4-oxadiazole oralmente activo para terapia de supressão de mutações sem sentido (ou nonsense)
PT11175569T PT2402002T (pt) 2005-04-08 2006-04-06 Composições de 1,2,4-oxadiazole oralmente activo para terapia de supressão de mutações nonsense

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PT06749439T PT1874306E (pt) 2005-04-08 2006-04-06 Composições de um 1,2,4-oxadiazole oralmente activo para terapia de supressão de mutações sem sentido (ou nonsense)

Country Status (16)

Country Link
US (7) US8716321B2 (pt)
EP (2) EP2402002B1 (pt)
JP (3) JP5800451B2 (pt)
CN (1) CN103720688A (pt)
AU (1) AU2006235115C1 (pt)
BR (1) BRPI0609089A2 (pt)
CA (1) CA2603402C (pt)
DK (2) DK1874306T3 (pt)
ES (2) ES2683359T3 (pt)
IL (2) IL186433A (pt)
MX (1) MX2007012206A (pt)
NO (2) NO339909B1 (pt)
NZ (1) NZ562197A (pt)
PL (2) PL2402002T3 (pt)
PT (2) PT1874306E (pt)
WO (1) WO2006110483A1 (pt)

Families Citing this family (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2887054T3 (es) 2003-04-11 2021-12-21 Ptc Therapeutics Inc Compuesto de ácido 1,2,4-oxadiazol benzoico y su uso para la supresión sin sentido y el tratamiento de enfermedades
CA2603402C (en) 2005-04-08 2017-10-31 Ptc Therapeutics, Inc. Compositions of an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy
IN2014CN04406A (pt) 2006-03-30 2015-09-04 Ptc Therapeutics Inc
US7645789B2 (en) 2006-04-07 2010-01-12 Vertex Pharmaceuticals Incorporated Indole derivatives as CFTR modulators
USRE50453E1 (en) 2006-04-07 2025-06-10 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
US7776905B2 (en) 2006-04-07 2010-08-17 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
PT2059513E (pt) 2006-09-08 2013-04-30 Ptc Therapeutics Inc Processo para a preparação de ácidos 1,2,4-oxadiazolebenzóicos
WO2008130370A1 (en) 2006-09-25 2008-10-30 Ptc Therapeutics, Inc. Hydroxylated 1,2,4-oxadiazole benzoic acid compounds, compositions thereof and the use for nonsense suppression
SG10201609581TA (en) 2006-09-25 2016-12-29 Ptc Therapeutics Inc Crystalline forms of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid
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
CL2007002923A1 (es) * 2006-10-12 2008-06-06 Ptc Therapeutics Inc Soc Organ Metodo para el tratamiento, prevencion o manejo de una enfermedad asociada con una mutacion sin sentido tal como cancer, diabetes o enfermedad pulmonar, que consiste en administrar a un paciente una cantidad efectiva de acido 3-[5-(2-fluoro-fenil)-[1
US8563573B2 (en) 2007-11-02 2013-10-22 Vertex Pharmaceuticals Incorporated Azaindole derivatives as CFTR modulators
GB0715087D0 (en) 2007-08-03 2007-09-12 Summit Corp Plc Drug combinations for the treatment of duchenne muscular dystrophy
TW200911237A (en) 2007-08-03 2009-03-16 Summit Corp Plc Drug combinations for the treatment of duchenne muscular dystrophy
HRP20140103T1 (hr) 2007-11-16 2014-05-09 Vertex Pharmaceuticals Inc. Izokinolinski modulatori transportera atp-vežuä†e kazete
HUE031913T2 (en) 2007-12-07 2017-08-28 Vertex Pharma Solid forms of 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl) benzoic acid
DK2615085T3 (en) 2008-03-31 2015-10-05 Vertex Pharma Pyridyl derivatives as CFTR modulators
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
LT2826776T (lt) 2010-03-25 2021-01-11 Vertex Pharmaceuticals Incorporated (r)-1(2,2-difluorbenzo(d)(1,3)dioksol-5-il)-n-(1-(2,3-dihidroksipropil)-6-fluor-2-(1-hidroksi-2-metilpropan-2-il)-1h-indol-5-il)-ciklopropankarboksamido kieta amorfinės formos dispersija
NZ602795A (en) 2010-04-07 2015-01-30 Vertex Pharma Solid forms of 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid
RS62767B1 (sr) 2010-04-07 2022-01-31 Vertex Pharma Farmaceutski sastavi 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioksol-5-il) ciklopropankarboksamido)-3-metilpiriodin-2-il)benzojeve kiseline i njihova primena
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
WO2012016930A1 (en) * 2010-08-05 2012-02-09 Universite De Droit Et Sante De Lille Compound useful for the treatment of nonsense-mutation-mediated diseases and pharmaceutical composition comprising said compound
US20120046330A1 (en) 2010-08-23 2012-02-23 Vertex Pharmaceuticals Incorporated 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
RU2640420C2 (ru) 2011-11-08 2018-01-09 Вертекс Фармасьютикалз Инкорпорейтед Модуляторы транспортеров атф-связывающей кассеты
MX355183B (es) 2012-01-25 2018-04-09 Vertex Pharma Formulaciones de acido 3- (6- (1- (2.2- difluorobenzo [d] [1,3] dioxol-5-il) ciclopropancarboxamido) -3- metilpiridin-2-il) benzoico.
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
SMT202500078T1 (it) 2012-11-02 2025-03-12 Vertex Pharma Composizioni farmaceutiche per il trattamento di malattie mediate da cftr
WO2014153643A1 (en) * 2013-03-26 2014-10-02 The University Of British Columbia Compositions and methods for use thereof in the treatment of aniridia
TWI695717B (zh) * 2014-03-06 2020-06-11 美商 Ptc 治療公司 １,２,４-二唑苯甲酸之鹽及醫藥組合物
JP6543268B2 (ja) 2014-04-15 2019-07-10 バーテックス ファーマシューティカルズ インコーポレイテッドＶｅｒｔｅｘ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｉｎｃｏｒｐｏｒａｔｅｄ 嚢胞性線維症膜コンダクタンス調節因子が媒介する疾患を処置するための医薬組成物
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