CL2016001215A1 - Tratamiento de hipercolesterolemia familiar homocigota. - Google Patents

Tratamiento de hipercolesterolemia familiar homocigota.

Info

Publication number
CL2016001215A1
CL2016001215A1 CL2016001215A CL2016001215A CL2016001215A1 CL 2016001215 A1 CL2016001215 A1 CL 2016001215A1 CL 2016001215 A CL2016001215 A CL 2016001215A CL 2016001215 A CL2016001215 A CL 2016001215A CL 2016001215 A1 CL2016001215 A1 CL 2016001215A1
Authority
CL
Chile
Prior art keywords
familial hypercholesterolemia
homozygous familial
treatment
hypercholesterolemia treatment
homozygous
Prior art date
Application number
CL2016001215A
Other languages
English (en)
Spanish (es)
Inventor
Charles A Mcwherter
Robert L Martin
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 CL2016001215A1 publication Critical patent/CL2016001215A1/es

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)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (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)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Endocrinology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Urology & Nephrology (AREA)
  • Emergency Medicine (AREA)
  • Vascular Medicine (AREA)
CL2016001215A 2013-11-20 2016-05-20 Tratamiento de hipercolesterolemia familiar homocigota. CL2016001215A1 (es)

Applications Claiming Priority (6)

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
US201461974785P 2014-04-03 2014-04-03
US201461974725P 2014-04-03 2014-04-03
US201461974816P 2014-04-03 2014-04-03

Publications (1)

Publication Number Publication Date
CL2016001215A1 true CL2016001215A1 (es) 2016-12-09

Family

ID=52014370

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2016001215A CL2016001215A1 (es) 2013-11-20 2016-05-20 Tratamiento de hipercolesterolemia familiar homocigota.

Country Status (14)

Country Link
US (1) US20150139987A1 (cg-RX-API-DMAC7.html)
EP (1) EP3071198A1 (cg-RX-API-DMAC7.html)
JP (1) JP2017505285A (cg-RX-API-DMAC7.html)
KR (1) KR20160079124A (cg-RX-API-DMAC7.html)
CN (1) CN105764498A (cg-RX-API-DMAC7.html)
AU (1) AU2014353246A1 (cg-RX-API-DMAC7.html)
CA (1) CA2930069A1 (cg-RX-API-DMAC7.html)
CL (1) CL2016001215A1 (cg-RX-API-DMAC7.html)
EA (1) EA201691039A1 (cg-RX-API-DMAC7.html)
HK (1) HK1224186A1 (cg-RX-API-DMAC7.html)
IL (1) IL245748A0 (cg-RX-API-DMAC7.html)
MX (1) MX2016006583A (cg-RX-API-DMAC7.html)
PH (1) PH12016500886A1 (cg-RX-API-DMAC7.html)
WO (1) WO2015077154A1 (cg-RX-API-DMAC7.html)

Families Citing this family (19)

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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
PT2929031T (pt) 2012-12-05 2018-01-19 Alnylam Pharmaceuticals Inc Composições de irna de pcsk9 e métodos de seu uso
US9624172B2 (en) 2014-02-17 2017-04-18 Hetero Research Foundation Polymorphs of lomitapide and its salts
CN106102734B (zh) 2014-03-20 2019-05-14 西玛贝医药公司 肝内胆汁淤积性疾病的治疗
US10272058B2 (en) 2014-03-20 2019-04-30 Cymabay Therapeutics, Inc. Treatment of intrahepatic cholestatic diseases
PL3129018T3 (pl) 2014-04-11 2020-05-18 Cymabay Therapeutics, Inc. Leczenie NAFLD i NASH
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
KR102793532B1 (ko) * 2015-08-25 2025-04-14 알닐람 파마슈티칼스 인코포레이티드 전구단백질 컨버타제 서브틸리신 켁신 (pcsk9) 유전자-관련 장애를 치료하기 위한 방법 및 조성물
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
JP7541810B2 (ja) 2016-03-03 2024-08-29 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ａｎｇｐｔｌ３阻害剤と組み合わせてｐｃｓｋ９阻害剤を投与することにより高脂血症を有する患者を処置するための方法
LT3448426T (lt) * 2016-04-28 2025-07-10 Regeneron Pharmaceuticals, Inc. Pacientų, sergančių šeimine hipercholesterolemija, gydymo būdai
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
WO2019101151A1 (zh) * 2017-11-23 2019-05-31 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
CN113710808A (zh) * 2018-12-20 2021-11-26 宾夕法尼亚州大学信托人 用于治疗家族性高胆固醇血症的基因疗法
US11248001B2 (en) 2019-01-18 2022-02-15 Astrazeneca Ab PCSK9 inhibitors and methods of use thereof
US20210145774A1 (en) 2019-11-14 2021-05-20 Cymabay Therapeutics, Inc. Treatment of alcoholic liver disease
US20210145775A1 (en) 2019-11-14 2021-05-20 Cymabay Therapeutics, Inc. Treatment of intestinal barrier dysfunction and associated diseases
CA3186856A1 (en) * 2020-07-29 2022-02-03 Ruth NALLEN Lomitapide for use in methods of treating hyperlipidemia and hypercholesterolemia in pediatric patients

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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
US7301050B2 (en) 2003-09-19 2007-11-27 Janssen Pharmaceutical N.V. 4-((phenoxyalkyl)thio)-phenoxyacetic acids and analogs
KR20060129082A (ko) 2004-03-05 2006-12-14 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 부작용을 최소화하면서 과지질혈증 및 과콜레스테롤혈증과연관된 질환 또는 질병의 치료 방법
JO3006B1 (ar) 2005-09-14 2016-09-05 Janssen Pharmaceutica Nv املاح ليسين مبتكرة من مشتقات حامض 4-((فينوكسي الكيل)ثيو) فينوكسي الخليك
ES2712052T3 (es) 2008-10-17 2019-05-09 Cymabay Therapeutics Inc Métodos para reducir partículas de LDL pequeñas y densas
JOP20200043A1 (ar) * 2011-05-10 2017-06-16 Amgen Inc طرق معالجة أو منع الاضطرابات المختصة بالكوليسترول

Also Published As

Publication number Publication date
IL245748A0 (en) 2016-07-31
EP3071198A1 (en) 2016-09-28
EA201691039A1 (ru) 2016-11-30
CN105764498A (zh) 2016-07-13
WO2015077154A1 (en) 2015-05-28
CA2930069A1 (en) 2015-05-28
PH12016500886A1 (en) 2016-06-20
JP2017505285A (ja) 2017-02-16
HK1224186A1 (zh) 2017-08-18
AU2014353246A1 (en) 2016-06-16
US20150139987A1 (en) 2015-05-21
KR20160079124A (ko) 2016-07-05
MX2016006583A (es) 2016-09-06

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