PL2600865T3 - Związek użyteczny do leczenia chorób, w których pośredniczy mutacja nonsensowna oraz kompozycje farmaceutyczne zawierające wymieniony związek - Google Patents

Związek użyteczny do leczenia chorób, w których pośredniczy mutacja nonsensowna oraz kompozycje farmaceutyczne zawierające wymieniony związek

Info

Publication number
PL2600865T3
PL2600865T3 PL11736401T PL11736401T PL2600865T3 PL 2600865 T3 PL2600865 T3 PL 2600865T3 PL 11736401 T PL11736401 T PL 11736401T PL 11736401 T PL11736401 T PL 11736401T PL 2600865 T3 PL2600865 T3 PL 2600865T3
Authority
PL
Poland
Prior art keywords
compound
nonsense
mutation
treatment
pharmaceutical composition
Prior art date
Application number
PL11736401T
Other languages
English (en)
Inventor
Fabrice Lejeune
Benoit Deprez
Terence Beghyn
Sara Sofia Gonzalez-Hilarion
Original Assignee
Institut National De La Sante Et De La Rech Medicale
Pasteur Institut
Univ De Lille
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 Institut National De La Sante Et De La Rech Medicale, Pasteur Institut, Univ De Lille filed Critical Institut National De La Sante Et De La Rech Medicale
Publication of PL2600865T3 publication Critical patent/PL2600865T3/pl

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K31/435Heterocyclic 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
    • A61K31/4353Heterocyclic 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 ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/436Heterocyclic 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 ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/12Ketones
    • A61K31/122Ketones having the oxygen directly attached to a ring, e.g. quinones, vitamin K1, anthralin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/335Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/35Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K31/435Heterocyclic 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
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    • A61P13/02Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
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    • AHUMAN NECESSITIES
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    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/08Antiepileptics; Anticonvulsants
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    • A61P25/28Drugs 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
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    • A61P27/16Otologicals
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P3/00Drugs for disorders of the metabolism
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/12Drugs for disorders of the metabolism for electrolyte homeostasis
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    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00Drugs for disorders of the endocrine system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00Drugs for disorders of the endocrine system
    • A61P5/14Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00Drugs for disorders of the endocrine system
    • A61P5/38Drugs for disorders of the endocrine system of the suprarenal hormones
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
PL11736401T 2010-08-05 2011-07-29 Związek użyteczny do leczenia chorób, w których pośredniczy mutacja nonsensowna oraz kompozycje farmaceutyczne zawierające wymieniony związek PL2600865T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
FR1056472 2010-08-05
PCT/EP2011/063126 WO2012016930A1 (en) 2010-08-05 2011-07-29 Compound useful for the treatment of nonsense-mutation-mediated diseases and pharmaceutical composition comprising said compound
EP11736401.8A EP2600865B1 (en) 2010-08-05 2011-07-29 Compound useful for the treatment of nonsense-mutation-mediated diseases and pharmaceutical composition comprising said compound

Publications (1)

Publication Number Publication Date
PL2600865T3 true PL2600865T3 (pl) 2019-05-31

Family

ID=43447136

Family Applications (1)

Application Number Title Priority Date Filing Date
PL11736401T PL2600865T3 (pl) 2010-08-05 2011-07-29 Związek użyteczny do leczenia chorób, w których pośredniczy mutacja nonsensowna oraz kompozycje farmaceutyczne zawierające wymieniony związek

Country Status (10)

Country Link
US (2) US9913829B2 (pl)
EP (1) EP2600865B1 (pl)
JP (1) JP5877200B2 (pl)
CN (1) CN103189060A (pl)
CA (1) CA2806825C (pl)
DK (1) DK2600865T3 (pl)
ES (1) ES2710854T3 (pl)
IL (1) IL224544B (pl)
PL (1) PL2600865T3 (pl)
WO (1) WO2012016930A1 (pl)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2659763T3 (es) 2011-02-14 2018-03-19 The Regents Of The University Of Michigan Composiciones y procedimientos para el tratamiento de obesidad y trastornos relacionados
CN107569487A (zh) 2012-12-13 2018-01-12 诺华股份有限公司 作为无义突变抑制子的嘧啶并[4,5‑b]喹啉‑4,5(3H,10H)‑二酮
US10987370B2 (en) * 2012-12-24 2021-04-27 Ramot At Tel-Aviv University Ltd. Methods of inducing read-through of a nonsense mutation associated with ataxia telangiectasia, Rett syndrome or spinal muscular atrophy by erythromycin or azithromycin
WO2014179734A1 (en) 2013-05-02 2014-11-06 The Regents Of The University Of Michigan Deuterated amlexanox
JP2017507930A (ja) * 2014-02-07 2017-03-23 ザ リージェンツ オブ ザ ユニヴァシティ オブ ミシガン IKKε/TBK1阻害剤とベータアドレナリン作動性アゴニストまたは交感神経系活性化剤との組合せ
AP2016009545A0 (en) * 2014-06-03 2016-11-30 Novartis Ag Naphthyridinedione derivatives
ES2743184T3 (es) * 2014-06-03 2020-02-18 Novartis Ag Derivados de pirimido[4,5-b]quinolin-4,5(3H,10H)-diona como supresores de mutaciones finalizadoras
ES2743183T3 (es) 2014-06-03 2020-02-18 Novartis Ag Derivados de piridopirimidinodiona como supresores de mutaciones finalizadoras
US20170196842A1 (en) * 2014-06-06 2017-07-13 Ptc Therapeutics, Inc. Methods of treatment using a 1,2,4-oxadiazole benzoic acid
WO2015198329A1 (en) 2014-06-25 2015-12-30 Bio Blast Pharma Ltd. Injectable formulations for intrathecal administration of antibiotic agents
WO2016073545A1 (en) 2014-11-06 2016-05-12 Concert Pharmaceuticals, Inc. Phenyloxadiazole benzoic acids
CN104450727A (zh) * 2014-11-13 2015-03-25 深圳华大基因科技有限公司 X-连锁低磷酸盐血症性佝偻病的致病基因及其编码蛋白质和应用
CA3003132A1 (en) * 2015-10-30 2017-05-04 Ptc Therapeutics, Inc. Methods for treating epilepsy
WO2017112955A1 (en) 2015-12-23 2017-06-29 Pharma Llc Moonshot Methods for inducing an immune response by inhibition of nonsense mediated decay
WO2017132538A1 (en) 2016-01-29 2017-08-03 The Regents Of The University Of Michigan Amlexanox analogs
WO2018050706A1 (en) * 2016-09-13 2018-03-22 Marco Cipolli Method of treatment of shwachman-diamond syndrome
FR3057776A1 (fr) 2016-10-21 2018-04-27 Museum National D'histoire Naturelle Lepista flaccida destine a etre utilise dans le traitement d'une maladie due a une mutation non-sens
FR3057774A1 (fr) 2016-10-21 2018-04-27 Museum National D'histoire Naturelle Derives de la purine pour leur utilisation dans le traitement ou la prevention de maladies dues a une mutation non-sens
US11654135B2 (en) 2017-06-22 2023-05-23 Moonshot Pharma Llc Methods for treating colon cancer with compositions comprising amlexanox and immune checkpoint inhibitors
CN109200053A (zh) * 2018-11-17 2019-01-15 王海玲 一种抗癫痫外用药物及其用途

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2770035T3 (es) * 2003-04-11 2020-06-30 Ptc Therapeutics Inc Compuesto de ácido 1,2,4-oxadiazol benzoico y su uso para la supresión sin sentido y el tratamiento de enfermedades
MX2007012206A (es) * 2005-04-08 2007-12-05 Ptc Therapeutics Inc Composiciones de 1,2,4-oxadiazol activo por via oral para terapia de supresion de mutacion interruptora.
US7772255B2 (en) 2005-12-13 2010-08-10 Supratek Pharma, Inc. Method of treating tumors with azaxanthones
WO2008021210A2 (en) 2006-08-11 2008-02-21 Combinatorx, Incorporated Methods and compositions for the treatment of neurodegenerative disorders
FR2912745A1 (fr) 2007-02-19 2008-08-22 Centre Nat Rech Scient Nouveaux composes derives d'indole et compositions pharmaceutiques les contenant
AU2008273997A1 (en) * 2007-07-11 2009-01-15 Cardoz Ab Combination for use in the treatment of atherosclerosis comprising a mast cell inhibitor and a PPAR gamma agonist
WO2009151569A2 (en) 2008-06-09 2009-12-17 Combinatorx, Incorporated Beta adrenergic receptor agonists for the treatment of b-cell proliferative disorders
WO2010062366A1 (en) * 2008-10-31 2010-06-03 Arginetix, Inc. Compositions and methods of treating endothelial disorders
WO2010139985A1 (en) 2009-06-01 2010-12-09 Biocopea Limited The use of amlexanox in the therapy of neutrophil-driven diseases

Also Published As

Publication number Publication date
EP2600865A1 (en) 2013-06-12
US10265302B2 (en) 2019-04-23
EP2600865B1 (en) 2018-11-14
US9913829B2 (en) 2018-03-13
JP2013535479A (ja) 2013-09-12
CA2806825A1 (en) 2012-02-09
US20180147191A1 (en) 2018-05-31
JP5877200B2 (ja) 2016-03-02
CN103189060A (zh) 2013-07-03
DK2600865T3 (en) 2019-02-25
CA2806825C (en) 2018-08-21
IL224544B (en) 2018-07-31
US20130217717A1 (en) 2013-08-22
ES2710854T3 (es) 2019-04-29
WO2012016930A1 (en) 2012-02-09

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