DK1874306T3 - Sammensætninger af en oralt aktiv 1,2,4-oxadiazol til nonsens-mutationssuppressionsterapi - Google Patents

Sammensætninger af en oralt aktiv 1,2,4-oxadiazol til nonsens-mutationssuppressionsterapi

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Publication number
DK1874306T3
DK1874306T3 DK06749439.3T DK06749439T DK1874306T3 DK 1874306 T3 DK1874306 T3 DK 1874306T3 DK 06749439 T DK06749439 T DK 06749439T DK 1874306 T3 DK1874306 T3 DK 1874306T3
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Denmark
Prior art keywords
oxadiazole
compositions
nonsense mutation
orally active
suppression therapy
Prior art date
Application number
DK06749439.3T
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
Application granted granted Critical
Publication of DK1874306T3 publication Critical patent/DK1874306T3/da

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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41Heterocyclic 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
    • A61K31/4245Oxadiazoles
    • AHUMAN NECESSITIES
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    • A61K9/00Medicinal preparations characterised by special physical form
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    • A61K9/141Intimate 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
    • A61K9/145Intimate 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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DK06749439.3T 2005-04-08 2006-04-06 Sammensætninger af en oralt aktiv 1,2,4-oxadiazol til nonsens-mutationssuppressionsterapi DK1874306T3 (da)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
DK1874306T3 true DK1874306T3 (da) 2012-10-01

Family

ID=36763092

Family Applications (2)

Application Number Title Priority Date Filing Date
DK11175569.0T DK2402002T3 (da) 2005-04-08 2006-04-06 Sammensætninger omfattende en 1,2,4-oxadiazol og anvendelser deraf til behandling af sygdomme associeret med et for tidligt stop-kodon
DK06749439.3T DK1874306T3 (da) 2005-04-08 2006-04-06 Sammensætninger af en oralt aktiv 1,2,4-oxadiazol til nonsens-mutationssuppressionsterapi

Family Applications Before (1)

Application Number Title Priority Date Filing Date
DK11175569.0T DK2402002T3 (da) 2005-04-08 2006-04-06 Sammensætninger omfattende en 1,2,4-oxadiazol og anvendelser deraf til behandling af sygdomme associeret med et for tidligt stop-kodon

Country Status (17)

Country Link
US (7) US8716321B2 (da)
EP (2) EP1874306B1 (da)
JP (3) JP5800451B2 (da)
CN (1) CN103720688A (da)
AU (1) AU2006235115C1 (da)
BR (1) BRPI0609089A2 (da)
CA (1) CA2603402C (da)
DK (2) DK2402002T3 (da)
ES (2) ES2683359T3 (da)
HK (1) HK1111897A1 (da)
IL (2) IL186433A (da)
MX (1) MX2007012206A (da)
NO (2) NO339909B1 (da)
NZ (1) NZ562197A (da)
PL (2) PL2402002T3 (da)
PT (2) PT2402002T (da)
WO (1) WO2006110483A1 (da)

Families Citing this family (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI3889142T1 (sl) 2003-04-11 2022-10-28 Ptc Therapeutics, Inc. Spojine 1,2,4-oksadiazol benzojske kisline in njihova uporaba za zatiranje nesmiselne ter zdravljenje bolezni
CN103720688A (zh) 2005-04-08 2014-04-16 Ptc医疗公司 用于无义突变抑制治疗的口服活性1,2,4-噁二唑组合物
SG170819A1 (en) 2006-03-30 2011-05-30 Ptc Therapeutics Inc Methods for the production of functional protein from dna having a nonsense mutation and the treatment of disorders associated therewith
US10022352B2 (en) 2006-04-07 2018-07-17 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
PT2674428T (pt) 2006-04-07 2016-07-14 Vertex Pharma Modeladores de transportadores de cassetes de ligação de atp
US7645789B2 (en) 2006-04-07 2010-01-12 Vertex Pharmaceuticals Incorporated Indole derivatives as CFTR modulators
PL2059513T3 (pl) * 2006-09-08 2013-06-28 Ptc Therapeutics Inc Sposób wytwarzania pochodnych 1,2,4-oksadiazolowych kwasu benzoesowego
US8101641B2 (en) 2006-09-25 2012-01-24 Ptc Therapeutics, Inc. Hydroxylated 1,2,4-oxadiazole benzoic acid compounds and compositions thereof
JP5714228B2 (ja) 2006-09-25 2015-05-07 ピーティーシー セラピューティクス,インコーポレーテッド 3−[5−(2−フルオロフェニル)−[1,2,4]オキサジアゾール−3−イル]−安息香酸の結晶形
DK2073805T3 (da) 2006-10-12 2015-06-15 Ptc Therapeutics Inc Fremgangsmåder til dosering af en oralt aktiv 1,2,4-oxadiazol til terapi for suppression af nonsens-mutation
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
EP2170396B1 (en) 2007-08-03 2016-12-21 Summit Corporation Plc Drug combinations for the treatment of duchenne muscular dystrophy
GB0715087D0 (en) 2007-08-03 2007-09-12 Summit Corp Plc Drug combinations for the treatment of duchenne muscular dystrophy
CA2705562C (en) 2007-11-16 2016-05-17 Vertex Pharmaceuticals Incorporated Isoquinoline modulators of atp-binding cassette transporters
WO2009073757A1 (en) 2007-12-07 2009-06-11 Vertex Pharmaceuticals Incorporated Solid forms of 3-(6-(1-(2,2-difluorobenzo[d][1,3] dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl) benzoic acid
ES2552990T3 (es) 2008-03-31 2015-12-03 Vertex Pharmaceuticals Incorporated Derivados de piridilo como moduladores del CFTR
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
SI2826776T1 (sl) 2010-03-25 2021-02-26 Vertex Pharmaceuticals Incorporated Trdna disperzija amorfne oblike (R)-1(2,2-difluorobenzo(D)(1,3)dioksol-5-il)-N-(1-(2,3-dihidroksipropil) -6-fluoro-2-(1-hidroksi-2-metilpropan-2-il)-1H-indol-5-il)- ciklopropankarboksamida
WO2011127241A2 (en) 2010-04-07 2011-10-13 Vertex Pharmaceuticals Incorporated Pharmaceutical compositions of 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyriodin-2-yl)benzoic acid and administration thereof
PL2555754T3 (pl) 2010-04-07 2016-09-30 Formy stałe kwasu 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioksol-5-ilo)cyklopropanokarboksyamido)-3-metylopirydyn-2-ylo)benzoesowego
US9035072B2 (en) 2010-04-22 2015-05-19 Vertex Pharmaceuticals Incorporated Process of producing cycloalkylcarboxamido-indole compounds
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
EP2600865B1 (en) 2010-08-05 2018-11-14 Université de Lille Compound useful for the treatment of nonsense-mutation-mediated diseases and pharmaceutical composition comprising said compound
AU2011293658B2 (en) 2010-08-23 2015-03-05 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 thereof
MX357328B (es) 2011-11-08 2018-07-05 Vertex Pharma Moduladores de trasportadores de casete enlazante de atp.
JP2015504920A (ja) 2012-01-25 2015-02-16 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 3−(6−(1−(2,2−ジフルオロベンゾ[d][1,3]ジオキソール−5−イル)シクロプロパンカルボキサミド)−3−メチルピリジン−2−イル)安息香酸の製剤
US9012496B2 (en) 2012-07-16 2015-04-21 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
JP6302923B2 (ja) 2012-11-02 2018-03-28 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 Cftrが媒介する疾患の処置のための医薬組成物
WO2014153643A1 (en) * 2013-03-26 2014-10-02 The University Of British Columbia Compositions and methods for use thereof in the treatment of aniridia
US9873677B2 (en) 2014-03-06 2018-01-23 Ptc Therapeutics, Inc. Pharmaceutical compositions and salts of a 1,2,4-oxadiazole benzoic acid
PL3424534T3 (pl) 2014-04-15 2021-11-22 Vertex Pharmaceuticals Incorporated Kompozycje farmaceutyczne do leczenia chorób, w których pośredniczy mukowiscydozowy przezbłonowy regulator przewodnictwa
TW201625241A (zh) * 2014-06-06 2016-07-16 Ptc治療公司 使用1,2,4-二唑苯甲酸之治療方法
EP4159717A1 (en) 2014-10-06 2023-04-05 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
CN108348527A (zh) 2015-10-30 2018-07-31 Ptc医疗公司 用于治疗癫痫的方法
CA3048202A1 (en) * 2015-12-23 2017-06-29 Moonshot Pharma Llc Methods for inducing an immune response by inhibition of nonsense mediated decay
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
US11207300B2 (en) 2016-09-13 2021-12-28 Marco Cipolli Method of treatment of Shwachman-diamond syndrome
US11654135B2 (en) 2017-06-22 2023-05-23 Moonshot Pharma Llc Methods for treating colon cancer with compositions comprising amlexanox and immune checkpoint inhibitors
EA202091063A1 (ru) 2017-11-01 2020-09-18 Бристол-Маерс Сквибб Компани Мостиковые бициклические соединения в качестве модуляторов фарнезоидного х-рецептора
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
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
SG11202108796YA (en) 2019-02-15 2021-09-29 Bristol Myers Squibb Co Substituted bicyclic compounds as farnesoid x receptor modulators
BR112021015688A2 (pt) 2019-02-15 2021-10-26 Bristol-Myers Squibb Company Compostos de amida substituídos úteis como moduladores do receptor do farnesoide x
KR20210129119A (ko) 2019-02-15 2021-10-27 브리스톨-마이어스 스큅 컴퍼니 파르네소이드 x 수용체 조정제로서 유용한 치환된 아미드 화합물
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
MXPA03011196A (es) * 2001-06-08 2004-10-28 Cytovia Inc 3-aril-5-aril-[1,2,4]-oxadiazoles sustituidos y analogos.
MY151199A (en) * 2001-11-02 2014-04-30 Rigel Pharmaceuticals Inc Substituted diphenyl heterocycles useful for treating hcv infection
BR0313266A (pt) * 2002-08-09 2005-06-21 Astrazeneca Ab Compostos, respectivos processos de preparação e uso, formulação farmacêutica e métodos de prevenção e/ou tratamento de um distúrbio mediado pelo receptor de mglur5 e de inibição da ativação de receptores de mglur5
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
SI3889142T1 (sl) * 2003-04-11 2022-10-28 Ptc Therapeutics, Inc. Spojine 1,2,4-oksadiazol benzojske kisline in njihova uporaba za zatiranje nesmiselne ter zdravljenje bolezni
US20050075375A1 (en) * 2003-05-14 2005-04-07 Anadys Pharmaceuticals, Inc. Heterocyclic compounds for treating hepatitis C virus
CN103720688A (zh) 2005-04-08 2014-04-16 Ptc医疗公司 用于无义突变抑制治疗的口服活性1,2,4-噁二唑组合物
US20070010553A1 (en) * 2005-07-01 2007-01-11 Astrazeneca Ab New use
PL2059513T3 (pl) 2006-09-08 2013-06-28 Ptc Therapeutics Inc Sposób wytwarzania pochodnych 1,2,4-oksadiazolowych kwasu benzoesowego
JP5714228B2 (ja) * 2006-09-25 2015-05-07 ピーティーシー セラピューティクス,インコーポレーテッド 3−[5−(2−フルオロフェニル)−[1,2,4]オキサジアゾール−3−イル]−安息香酸の結晶形
US9873677B2 (en) 2014-03-06 2018-01-23 Ptc Therapeutics, Inc. Pharmaceutical compositions and salts of a 1,2,4-oxadiazole benzoic acid

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