PH12016500886A1 - Treatment of homozygous familial hypercholesterolemia - Google Patents

Treatment of homozygous familial hypercholesterolemia

Info

Publication number
PH12016500886A1
PH12016500886A1 PH12016500886A PH12016500886A PH12016500886A1 PH 12016500886 A1 PH12016500886 A1 PH 12016500886A1 PH 12016500886 A PH12016500886 A PH 12016500886A PH 12016500886 A PH12016500886 A PH 12016500886A PH 12016500886 A1 PH12016500886 A1 PH 12016500886A1
Authority
PH
Philippines
Prior art keywords
treatment
familial hypercholesterolemia
homozygous familial
inhibitor
homozygous
Prior art date
Application number
PH12016500886A
Other languages
English (en)
Inventor
Robert L Martin
Charles A Mcwherter
Patrick J O'mara
Original Assignee
Cymabay 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 Cymabay Therapeutics Inc filed Critical Cymabay Therapeutics Inc
Publication of PH12016500886A1 publication Critical patent/PH12016500886A1/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A61K31/192—Carboxylic acids, e.g. valproic acid having aromatic groups, e.g. sulindac, 2-aryl-propionic acids, ethacrynic acid 
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K31/435—Heterocyclic 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
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A61K31/4468—Non condensed piperidines, e.g. piperocaine having a nitrogen directly attached in position 4, e.g. clebopride, fentanyl
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/40—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Diabetes (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Endocrinology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Emergency Medicine (AREA)
PH12016500886A 2013-11-20 2016-05-12 Treatment of homozygous familial hypercholesterolemia PH12016500886A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201361906837P 2013-11-20 2013-11-20
US201461942438P 2014-02-20 2014-02-20
US201461942941P 2014-02-21 2014-02-21
US201461974816P 2014-04-03 2014-04-03
US201461974785P 2014-04-03 2014-04-03
US201461974725P 2014-04-03 2014-04-03
PCT/US2014/065742 WO2015077154A1 (en) 2013-11-20 2014-11-14 Treatment of homozygous familial hypercholesterolemia

Publications (1)

Publication Number Publication Date
PH12016500886A1 true PH12016500886A1 (en) 2016-06-20

Family

ID=52014370

Family Applications (1)

Application Number Title Priority Date Filing Date
PH12016500886A PH12016500886A1 (en) 2013-11-20 2016-05-12 Treatment of homozygous familial hypercholesterolemia

Country Status (14)

Country Link
US (1) US20150139987A1 (enExample)
EP (1) EP3071198A1 (enExample)
JP (1) JP2017505285A (enExample)
KR (1) KR20160079124A (enExample)
CN (1) CN105764498A (enExample)
AU (1) AU2014353246A1 (enExample)
CA (1) CA2930069A1 (enExample)
CL (1) CL2016001215A1 (enExample)
EA (1) EA201691039A1 (enExample)
HK (1) HK1224186A1 (enExample)
IL (1) IL245748A0 (enExample)
MX (1) MX2016006583A (enExample)
PH (1) PH12016500886A1 (enExample)
WO (1) WO2015077154A1 (enExample)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9255154B2 (en) 2012-05-08 2016-02-09 Alderbio Holdings, Llc Anti-PCSK9 antibodies and use thereof
WO2014089313A1 (en) 2012-12-05 2014-06-12 Alnylam Pharmaceuticals PCSK9 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
US9624172B2 (en) 2014-02-17 2017-04-18 Hetero Research Foundation Polymorphs of lomitapide and its salts
PL3119384T3 (pl) 2014-03-20 2019-03-29 Cymabay Therapeutics, Inc. Leczenie wewnątrzwątrobowych chorób cholestatycznych
US10272058B2 (en) 2014-03-20 2019-04-30 Cymabay Therapeutics, Inc. Treatment of intrahepatic cholestatic diseases
CN106163508A (zh) 2014-04-11 2016-11-23 西玛贝医药公司 Nafld和nash的治疗
BR102015025502B1 (pt) * 2015-04-30 2022-06-21 Aegerion Pharmaceuticals, Inc Composição de lomitapida, tablete, produto de lomitapida, métodos para analisar uma composição de amostra de lomitapida e para determinar uma quantidade de uma impureza em uma amostra da composição
CN114939124A (zh) * 2015-08-25 2022-08-26 阿尔尼拉姆医药品有限公司 治疗前蛋白转化酶枯草杆菌蛋白酶kexin(pcsk9)基因相关障碍的方法和组合物
CN105481758A (zh) * 2016-01-13 2016-04-13 天津药物研究院有限公司 一种洛美他派晶型ⅰ及其制备方法和用途
MA43734A (fr) 2016-03-03 2018-11-28 Regeneron Pharma Procédés de traitement de patients atteints d'hyperlipidémie par administration d'un inhibiteur de pcsk9 en combinaison avec un inhibiteur d'angptl3
CN109069641A (zh) * 2016-04-28 2018-12-21 瑞泽恩制药公司 用于治疗家族性高胆固醇血症患者的方法
WO2017200715A1 (en) * 2016-05-19 2017-11-23 Cymabay Therapeutics, Inc. Treatment of severe hyperlipidemia
US10512622B2 (en) 2017-07-14 2019-12-24 Cymabay Therapeutics, Inc. Treatment of intrahepatic cholestatic diseases
CN111836620B (zh) * 2017-11-23 2023-11-03 浙江海正药业股份有限公司 一种海泽麦布和HMG-CoA还原酶抑制剂的药物组合物
CA3123844A1 (en) * 2018-12-20 2020-06-25 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
EP4470609A3 (en) 2019-01-18 2025-03-12 Astrazeneca AB Pcsk9 inhibitors and methods of use thereof
US20210145775A1 (en) 2019-11-14 2021-05-20 Cymabay Therapeutics, Inc. Treatment of intestinal barrier dysfunction and associated diseases
WO2021097027A1 (en) 2019-11-14 2021-05-20 Cymabay Therapeutics, Inc. Seladelpar for use in the treatment of alcoholic liver disease
CA3186856A1 (en) 2020-07-29 2022-02-03 Ruth NALLEN Lomitapide for use in methods of treating hyperlipidemia and hypercholesterolemia in pediatric patients

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5739135A (en) 1993-09-03 1998-04-14 Bristol-Myers Squibb Company Inhibitors of microsomal triglyceride transfer protein and method
US5883109A (en) 1996-07-24 1999-03-16 Bristol-Myers Squibb Company Method for lowering serum lipid levels employing an MTP inhibitor in combination with another cholesterol lowering drug
US6066653A (en) 1997-01-17 2000-05-23 Bristol-Myers Squibb Co. Method of treating acid lipase deficiency diseases with an MTP inhibitor and cholesterol lowering drugs
US7407943B2 (en) 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
PL1667964T3 (pl) 2003-09-19 2010-01-29 Janssen Pharmaceutica Nv Kwasy 4-((fenoksyalkilo)tio)-fenoksyoctowe i analogi
ES2399721T5 (es) 2004-03-05 2016-05-25 Univ Pennsylvania Métodos para tratar trastornos o enfermedades asociados a la hiperlipidemia e hipercolesterolemia minimizando los efectos adversos
JO3006B1 (ar) 2005-09-14 2016-09-05 Janssen Pharmaceutica Nv املاح ليسين مبتكرة من مشتقات حامض 4-((فينوكسي الكيل)ثيو) فينوكسي الخليك
WO2010045361A1 (en) * 2008-10-17 2010-04-22 Metabolex, Inc. Methods of reducing small, dense ldl particles
JOP20200043A1 (ar) * 2011-05-10 2017-06-16 Amgen Inc طرق معالجة أو منع الاضطرابات المختصة بالكوليسترول

Also Published As

Publication number Publication date
CL2016001215A1 (es) 2016-12-09
IL245748A0 (en) 2016-07-31
AU2014353246A1 (en) 2016-06-16
HK1224186A1 (zh) 2017-08-18
EP3071198A1 (en) 2016-09-28
CN105764498A (zh) 2016-07-13
EA201691039A1 (ru) 2016-11-30
CA2930069A1 (en) 2015-05-28
JP2017505285A (ja) 2017-02-16
US20150139987A1 (en) 2015-05-21
MX2016006583A (es) 2016-09-06
KR20160079124A (ko) 2016-07-05
WO2015077154A1 (en) 2015-05-28

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