JP6849686B2 - 重水素化されたcftr増強剤の投与 - Google Patents

重水素化されたcftr増強剤の投与 Download PDF

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JP6849686B2
JP6849686B2 JP2018534496A JP2018534496A JP6849686B2 JP 6849686 B2 JP6849686 B2 JP 6849686B2 JP 2018534496 A JP2018534496 A JP 2018534496A JP 2018534496 A JP2018534496 A JP 2018534496A JP 6849686 B2 JP6849686 B2 JP 6849686B2
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JP2018534348A (ja
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バージニア ブラマン,
バージニア ブラマン,
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バーテックス ファーマシューティカルズ (ヨーロッパ) リミテッド
バーテックス ファーマシューティカルズ (ヨーロッパ) リミテッド
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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
    • A61K31/47Quinolines; Isoquinolines
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • 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
    • A61K9/146Intimate 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 macromolecular compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/20Pills, tablets, discs, rods
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/20Pills, tablets, discs, rods
    • A61K9/2004Excipients; Inactive ingredients
    • A61K9/2009Inorganic compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/20Pills, tablets, discs, rods
    • A61K9/2004Excipients; Inactive ingredients
    • A61K9/2013Organic compounds, e.g. phospholipids, fats
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/20Pills, tablets, discs, rods
    • A61K9/2004Excipients; Inactive ingredients
    • A61K9/2013Organic compounds, e.g. phospholipids, fats
    • A61K9/2018Sugars, or sugar alcohols, e.g. lactose, mannitol; Derivatives thereof, e.g. polysorbates
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/20Pills, tablets, discs, rods
    • A61K9/2004Excipients; Inactive ingredients
    • A61K9/2022Organic macromolecular compounds
    • A61K9/205Polysaccharides, e.g. alginate, gums; Cyclodextrin
    • A61K9/2054Cellulose; Cellulose derivatives, e.g. hydroxypropyl methylcellulose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • A61P11/12Mucolytics
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D215/00Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems
    • C07D215/02Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen atoms or carbon atoms directly attached to the ring nitrogen atom
    • C07D215/16Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen atoms or carbon atoms directly attached to the ring nitrogen atom with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D215/48Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen
    • C07D215/54Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen attached in position 3
    • C07D215/56Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen attached in position 3 with oxygen atoms in position 4

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Pulmonology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Inorganic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
JP2018534496A 2015-09-21 2016-09-21 重水素化されたcftr増強剤の投与 Active JP6849686B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201562221531P 2015-09-21 2015-09-21
US62/221,531 2015-09-21
US201562238511P 2015-10-07 2015-10-07
US62/238,511 2015-10-07
US201662348855P 2016-06-10 2016-06-10
US62/348,855 2016-06-10
PCT/US2016/052922 WO2017053455A1 (en) 2015-09-21 2016-09-21 Administration of deuterated cftr potentiators

Related Child Applications (1)

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JP2021034591A Division JP7196217B2 (ja) 2015-09-21 2021-03-04 重水素化されたcftr増強剤の投与

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JP2018534348A JP2018534348A (ja) 2018-11-22
JP2018534348A5 JP2018534348A5 (https=) 2019-11-07
JP6849686B2 true JP6849686B2 (ja) 2021-03-24

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JP2021034591A Active JP7196217B2 (ja) 2015-09-21 2021-03-04 重水素化されたcftr増強剤の投与

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US (2) US20180353500A1 (https=)
EP (2) EP3352757B1 (https=)
JP (2) JP6849686B2 (https=)
KR (1) KR102828163B1 (https=)
CN (1) CN108367002A (https=)
AU (2) AU2016326441B2 (https=)
BR (1) BR112018005454A2 (https=)
CA (1) CA2998911C (https=)
HK (1) HK1258472A1 (https=)
IL (2) IL257993A (https=)
MA (1) MA42950A (https=)
MX (1) MX388470B (https=)
RU (1) RU2761344C2 (https=)
SG (1) SG10201913588YA (https=)
UA (1) UA124619C2 (https=)
WO (1) WO2017053455A1 (https=)

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HUE047354T2 (hu) 2011-05-18 2020-04-28 Vertex Pharmaceuticals Europe Ltd Ivacaftor deuterizált származékai
JP2015511583A (ja) 2012-02-27 2015-04-20 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 薬学的組成物およびその投与
MA41051B1 (fr) 2014-10-06 2020-11-30 Vertex Pharma Modulateurs du régulateur de conductance transmembranaire de la mucoviscidose
EP3277647A1 (en) 2015-03-31 2018-02-07 Vertex Pharmaceuticals (Europe) Limited Deuterated vx-661
KR102828163B1 (ko) 2015-09-21 2025-07-02 버텍스 파마슈티칼스 (유럽) 리미티드 중수소화된 cftr 강화제의 투여
EP3352758A2 (en) 2015-09-25 2018-08-01 Vertex Pharmaceuticals (Europe) Limited Deuterated cftr potentiators
CA3019380A1 (en) 2016-03-31 2017-10-05 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
CN109803962B (zh) 2016-09-30 2022-04-29 弗特克斯药品有限公司 囊性纤维化跨膜传导调控蛋白的调节剂、以及药物组合物
MD3551622T2 (ro) 2016-12-09 2021-03-31 Vertex Pharma Modulator al regulatorului conductanței transmembranare în fibroză chistică, compoziții farmaceutice, metode de tratament și procedeu pentru fabricarea modulatorului
US11253509B2 (en) 2017-06-08 2022-02-22 Vertex Pharmaceuticals Incorporated Methods of treatment for cystic fibrosis
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MA49631A (fr) 2017-07-17 2020-05-27 Vertex Pharma Méthodes de traitement de la fibrose kystique
JP7121794B2 (ja) 2017-08-02 2022-08-18 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 ピロリジン化合物を調製するためのプロセス
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MA51206A (fr) 2017-12-01 2020-10-07 Vertex Pharma Procédés pour préparer des modulateurs de régulateur de conductance transmembranaire de mucoviscidose
EP3720849A2 (en) 2017-12-08 2020-10-14 Vertex Pharmaceuticals Incorporated Processes for making modulators of cystic fibrosis transmembrane conductance regulator
TWI810243B (zh) 2018-02-05 2023-08-01 美商維泰克斯製藥公司 用於治療囊腫纖化症之醫藥組合物
JP7214743B2 (ja) 2018-02-15 2023-01-30 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 嚢胞性線維症膜コンダクタンス制御因子のモジュレーターとしての大環状分子、それらの医薬組成物、嚢胞性線維症の治療におけるそれらの使用、及びそれらの製造方法
EP3774825A1 (en) 2018-04-13 2021-02-17 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator, pharmaceutical compositions, methods of treatment, and process for making the modulator
EA202191361A1 (ru) 2018-11-14 2021-08-11 Вертекс Фармасьютикалз Инкорпорейтед Способы лечения муковисцидоза
UY38630A (es) 2019-04-03 2020-10-30 Vertex Pharma Agentes moduladores del regulador de la conductancia transmembrana de la fibrosis quística
WO2020214921A1 (en) 2019-04-17 2020-10-22 Vertex Pharmaceuticals Incorporated Solid forms of modulators of cftr
FR3096890B1 (fr) 2019-06-07 2021-05-14 H4 Orphan Pharma Utilisation d’une molécule opioïde pour traiter l’œil sec et l’œil allergique.
US20220313698A1 (en) 2019-08-14 2022-10-06 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
WO2021030552A1 (en) 2019-08-14 2021-02-18 Vertex Pharmaceuticals Incorporated Crystalline forms of cftr modulators
TWI899097B (zh) 2019-08-14 2025-10-01 美商維泰克斯製藥公司 製備cftr調節劑之方法
TWI867024B (zh) 2019-08-14 2024-12-21 美商維泰克斯製藥公司 囊腫纖維化跨膜傳導調節蛋白之調節劑
FR3109524B1 (fr) 2020-04-22 2022-04-08 H4 Orphan Pharma Utilisation d’un ligand multifonctionnel pour traiter l’œil sec et les dysfonctionnements des glandes de Meibomius et des glandes lacrymales.
FR3111071B1 (fr) 2020-06-04 2023-01-13 H4 Orphan Pharma Utilisation de ligands multifonctionnels pour traiter les syndromes de détresse respiratoire et le choc cytokinique liés aux infections virales à coronavirus.
CR20230120A (es) 2020-08-07 2023-09-01 Vertex Pharma Moduladores del regulador de la conductancia transmembrana de la fibrosis quística
IL300413A (en) 2020-08-13 2023-04-01 Vertex Pharma Crystal forms of CFTR modulators
WO2022076622A2 (en) 2020-10-07 2022-04-14 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
KR20230104618A (ko) 2020-10-07 2023-07-10 버텍스 파마슈티칼스 인코포레이티드 낭성 섬유증 막관통 전도도 조절자의 조절제
EP4225446A1 (en) 2020-10-07 2023-08-16 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
EP4225737A1 (en) 2020-10-07 2023-08-16 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
WO2022076626A1 (en) 2020-10-07 2022-04-14 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
AU2021356651A1 (en) 2020-10-07 2023-05-18 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
US20230373974A1 (en) 2020-10-07 2023-11-23 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
US20230373935A1 (en) 2020-10-07 2023-11-23 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
EP4225762A1 (en) 2020-10-07 2023-08-16 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
US12324802B2 (en) 2020-11-18 2025-06-10 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
KR20230118123A (ko) 2020-12-10 2023-08-10 버텍스 파마슈티칼스 인코포레이티드 낭성 섬유증 치료 방법
JP2025506382A (ja) 2022-02-03 2025-03-11 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 嚢胞性線維症の治療方法
JP2025505643A (ja) 2022-02-08 2025-02-28 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 嚢胞性線維症膜コンダクタンス制御因子の調節因子
CA3258118A1 (en) 2022-04-06 2023-10-12 Vertex Pharmaceuticals Incorporated MODULATORS OF THE TRANSMEMBRANE CONDUCTANCE REGULATOR IN CYSTIC FIBROSIS
WO2023224931A1 (en) 2022-05-16 2023-11-23 Vertex Pharmaceuticals Incorporated Methods of treatment for cystic fibrosis
WO2025076240A1 (en) 2023-10-04 2025-04-10 Vertex Pharmaceuticals Incorporated Solid forms of modulators of cystic fibrosis transmembrane conductance regulator
WO2025076235A1 (en) 2023-10-04 2025-04-10 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
WO2025166342A1 (en) 2024-02-04 2025-08-07 Vertex Pharmaceuticals Incorporated Combination of vanzacaftor, tezacaftor, deutivacaftor for use in treating cystic fibrosis
EP4648753B1 (en) 2024-02-07 2026-02-18 Vertex Pharmaceuticals Incorporated Compositions for the treatment of cftr-mediated diseases

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AU2011255237A1 (en) 2010-05-20 2012-11-29 Vertex Pharmaceuticals Incorporated Pharmaceutical compositions and administrations thereof
HUE047354T2 (hu) 2011-05-18 2020-04-28 Vertex Pharmaceuticals Europe Ltd Ivacaftor deuterizált származékai
RS56096B1 (sr) * 2011-05-18 2017-10-31 Concert Pharmaceuticals Inc Deuterisani derivati ivakaftora
JP2015511583A (ja) 2012-02-27 2015-04-20 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 薬学的組成物およびその投与
MY183582A (en) * 2012-11-19 2021-02-26 Vertex Pharmaceuticals Europe Ltd Deuterated cftr potentiators
CN103833630B (zh) * 2012-11-21 2018-11-13 顶点制药(欧洲)有限公司 氘化cftr增效剂
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KR102828163B1 (ko) 2015-09-21 2025-07-02 버텍스 파마슈티칼스 (유럽) 리미티드 중수소화된 cftr 강화제의 투여

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SG10201913588YA (en) 2020-02-27
MX2018003331A (es) 2018-08-16
MX388470B (es) 2025-03-20
RU2018114447A3 (https=) 2020-02-21
HK1258472A1 (zh) 2019-11-15
AU2016326441A1 (en) 2018-04-19
IL257993A (en) 2018-05-31
CA2998911A1 (en) 2017-03-30
RU2018114447A (ru) 2019-10-23
EP3352757C0 (en) 2023-08-16
EP4292588A2 (en) 2023-12-20
JP2018534348A (ja) 2018-11-22
JP2021091723A (ja) 2021-06-17
AU2022201135A1 (en) 2022-03-17
RU2761344C2 (ru) 2021-12-07
EP3352757A4 (en) 2018-08-01
UA124619C2 (uk) 2021-10-20
US20180353500A1 (en) 2018-12-13
EP3352757A1 (en) 2018-08-01
BR112018005454A2 (pt) 2018-10-09
AU2022201135B2 (en) 2024-03-28
CA2998911C (en) 2023-05-23
US20200375973A1 (en) 2020-12-03
CN108367002A (zh) 2018-08-03
EP4292588A3 (en) 2024-02-28
KR20180058745A (ko) 2018-06-01
KR102828163B1 (ko) 2025-07-02
WO2017053455A1 (en) 2017-03-30
MA42950A (fr) 2018-08-01
NZ741069A (en) 2024-12-20
AU2016326441B2 (en) 2021-11-25
JP7196217B2 (ja) 2022-12-26
EP3352757B1 (en) 2023-08-16
IL291517A (en) 2022-05-01

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