CA3079259A1 - Ganaxolone for use in treating genetic epileptic disorders - Google Patents

Ganaxolone for use in treating genetic epileptic disorders Download PDF

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Publication number
CA3079259A1
CA3079259A1 CA3079259A CA3079259A CA3079259A1 CA 3079259 A1 CA3079259 A1 CA 3079259A1 CA 3079259 A CA3079259 A CA 3079259A CA 3079259 A CA3079259 A CA 3079259A CA 3079259 A1 CA3079259 A1 CA 3079259A1
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CA
Canada
Prior art keywords
ganaxolone
day
neurosteroid
less
optionally substituted
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3079259A
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English (en)
French (fr)
Inventor
Lorianne K. Masuoka
Jaakko Lappalainen
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Immedica Pharma US Inc
Original Assignee
Marinus Pharmaceuticals 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 Marinus Pharmaceuticals Inc filed Critical Marinus Pharmaceuticals Inc
Publication of CA3079259A1 publication Critical patent/CA3079259A1/en
Pending legal-status Critical Current

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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
    • A61K31/56Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/565Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids not substituted in position 17 beta by a carbon atom, e.g. estrane, estradiol
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/56Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/12Carboxylic acids; Salts or anhydrides thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/24Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/34Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
    • 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
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0087Galenical forms not covered by A61K9/02 - A61K9/7023
    • A61K9/0095Drinks; Beverages; Syrups; Compositions for reconstitution thereof, e.g. powders or tablets to be dispersed in a glass of water; Veterinary drenches
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/08Solutions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/4841Filling excipients; Inactive ingredients
    • A61K9/4858Organic compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/4841Filling excipients; Inactive ingredients
    • A61K9/4866Organic macromolecular compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/08Antiepileptics; Anticonvulsants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/36Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
    • A61K47/38Cellulose; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/4841Filling excipients; Inactive ingredients
    • A61K9/485Inorganic compounds

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Engineering & Computer Science (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Pain & Pain Management (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Dermatology (AREA)
  • Molecular Biology (AREA)
  • Inorganic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Steroid Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3079259A 2017-11-10 2018-11-09 Ganaxolone for use in treating genetic epileptic disorders Pending CA3079259A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762584403P 2017-11-10 2017-11-10
US62/584,403 2017-11-10
PCT/US2018/060037 WO2019094724A1 (en) 2017-11-10 2018-11-09 Ganaxolone for use in treating genetic epileptic disoders

Publications (1)

Publication Number Publication Date
CA3079259A1 true CA3079259A1 (en) 2019-05-16

Family

ID=66438123

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3079259A Pending CA3079259A1 (en) 2017-11-10 2018-11-09 Ganaxolone for use in treating genetic epileptic disorders

Country Status (11)

Country Link
US (3) US20190160078A1 (https=)
EP (1) EP3706755A4 (https=)
JP (3) JP7312169B2 (https=)
KR (1) KR20200085837A (https=)
CN (1) CN111565724A (https=)
AU (2) AU2018364659A1 (https=)
CA (1) CA3079259A1 (https=)
EA (1) EA202091144A1 (https=)
MY (1) MY204493A (https=)
SG (2) SG10202110563YA (https=)
WO (1) WO2019094724A1 (https=)

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CA2882708A1 (en) 2012-08-21 2014-02-27 Sage Therapeutics, Inc. Methods of treating epilepsy or status epilepticus
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EP2986624B1 (en) 2013-04-17 2020-03-25 Sage Therapeutics, Inc. 19-nor neuroactive steroids for methods of treatment
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US9725481B2 (en) 2013-04-17 2017-08-08 Sage Therapeutics, Inc. 19-nor C3, 3-disubstituted C21-C-bound heteroaryl steroids and methods of use thereof
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RS61530B1 (sr) 2015-01-26 2021-04-29 Sage Therapeutics Inc Kompozicije i postupci za lečenje poremećaja cns
EP4155314A1 (en) 2015-02-20 2023-03-29 Sage Therapeutics, Inc. Neuroactive steroids, compositions, and uses thereof
CA2991311C (en) 2015-07-06 2025-05-06 Sage Therapeutics, Inc. OXYSTEROLS AND THEIR METHODS OF USE
EP3828194B1 (en) 2015-07-06 2025-04-16 Sage Therapeutics, Inc. Oxysterols and methods of use thereof
HUE071964T2 (hu) 2015-07-06 2025-10-28 Sage Therapeutics Inc Oxiszterolok és alkalmazási eljárásaik
KR20240157128A (ko) 2016-03-08 2024-10-31 세이지 테라퓨틱스, 인크. 신경활성 스테로이드, 조성물, 및 그의 용도
SMT202200300T1 (it) 2016-04-01 2022-09-14 Sage Therapeutics Inc Ossisteroli e relativi metodi d'uso
WO2017193046A1 (en) 2016-05-06 2017-11-09 Sage Therapeutics, Inc. Oxysterols and methods of use thereof
ES2884071T3 (es) 2016-07-07 2021-12-10 Sage Therapeutics Inc 24-hidroxiesteroles sustituidos en 11 para el tratamiento de afecciones relacionadas con NMDA
WO2018013615A1 (en) 2016-07-11 2018-01-18 Sage Therapeutics, Inc. C7, c12, and c16 substituted neuroactive steroids and their methods of use
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EP3504189A1 (en) 2016-08-23 2019-07-03 Sage Therapeutics, Inc. A crystalline 19-nor c3, 3-disubstituted c21-n-pyrazolyl steroid
ES2935057T3 (es) 2016-09-30 2023-03-01 Sage Therapeutics Inc C7 oxisteroles sustituidos y estos compuestos para su uso como moduladores de la NMDA
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Also Published As

Publication number Publication date
EA202091144A1 (ru) 2020-09-16
US20250082651A1 (en) 2025-03-13
AU2024227357A1 (en) 2024-10-31
JP2021502403A (ja) 2021-01-28
AU2018364659A1 (en) 2020-05-28
SG10202110563YA (en) 2021-11-29
US20220249515A1 (en) 2022-08-11
WO2019094724A1 (en) 2019-05-16
JP2025118661A (ja) 2025-08-13
SG11202004329TA (en) 2020-06-29
JP2023153783A (ja) 2023-10-18
MY204493A (en) 2024-08-30
KR20200085837A (ko) 2020-07-15
EP3706755A4 (en) 2021-11-10
EP3706755A1 (en) 2020-09-16
CN111565724A (zh) 2020-08-21
US20190160078A1 (en) 2019-05-30
JP7312169B2 (ja) 2023-07-20
WO2019094724A8 (en) 2022-10-06

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