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 [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/40—Immunoglobulins [IG], 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)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Molecular Biology (AREA)
  • Diabetes (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Endocrinology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Emergency Medicine (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (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
US201461974816P 2014-04-03 2014-04-03
US201461974725P 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 (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 (20)

* 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
KR102096014B1 (ko) 2012-12-05 2020-04-03 알닐람 파마슈티칼스 인코포레이티드 PCSK9 iRNA 조성물 및 그 사용 방법
WO2015121877A2 (en) * 2014-02-17 2015-08-20 Hetero Research Foundation Polymorphs of lomitapide and its salts
US10272058B2 (en) 2014-03-20 2019-04-30 Cymabay Therapeutics, Inc. Treatment of intrahepatic cholestatic diseases
HRP20181914T1 (hr) 2014-03-20 2019-01-11 Cymabay Therapeutics, Inc. Liječenje intrahepatičnih kolestatskih bolesti
SG11201608077PA (en) 2014-04-11 2016-10-28 Cymabay Therapeutics Inc Treatment of nafld and 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
MX2018002158A (es) * 2015-08-25 2018-07-06 Alnylam Pharmaceuticals Inc Mí‰TODOS Y COMPOSICIONES PARA TRATAR UN TRASTORNO ASOCIADO A GEN DE PROPROTEINA CONVERTASA SUBTILISINA KEXINA (PCSK9).
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
MX2018010401A (es) 2016-03-03 2019-03-06 Regeneron Pharma Metodos para el tratamiento de pacientes con hiperlipidemia administrando un inhibidor de pcsk9 en combinacion con un inhibidor de angptl3.
AU2017258105B2 (en) * 2016-04-28 2024-09-05 Regeneron Pharmaceuticals, Inc. Methods for treating patients with familial hypercholesterolemia
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
CN117085011A (zh) * 2017-11-23 2023-11-21 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
AU2019401638A1 (en) * 2018-12-20 2021-06-24 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
US11248001B2 (en) 2019-01-18 2022-02-15 Astrazeneca Ab PCSK9 inhibitors and methods of use thereof
US12559551B2 (en) 2019-05-24 2026-02-24 Regeneron Pharmaceuticals, Inc. Stabilized formulations containing anti-ANGPTL3 antibodies
US20210145775A1 (en) 2019-11-14 2021-05-20 Cymabay Therapeutics, Inc. Treatment of intestinal barrier dysfunction and associated diseases
US20210145774A1 (en) 2019-11-14 2021-05-20 Cymabay Therapeutics, Inc. Treatment of alcoholic liver disease
EP4188372A1 (en) * 2020-07-29 2023-06-07 Amryt Pharmaceuticals Inc. 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
EP2100877B1 (en) 2003-09-19 2012-01-25 Janssen Pharmaceutica N.V. 4-((Phenoxyalkyl)thio)-phenoxyacetic acids and analogs
NZ549721A (en) 2004-03-05 2010-08-27 Univ Pennsylvania Methods for treating disorders or diseases associated with hyperlipidemia and hypercholesterolemia while minimizing side-effects
JO3006B1 (ar) 2005-09-14 2016-09-05 Janssen Pharmaceutica Nv املاح ليسين مبتكرة من مشتقات حامض 4-((فينوكسي الكيل)ثيو) فينوكسي الخليك
BRPI0920170A2 (pt) * 2008-10-17 2016-06-21 Metabolex Inc métodos para diminuir a quantidade de partículas ldl pequenas e densas e de níveis de triglicerídeos e ldl no sangue em um humano, e para identificar um indivíduo candidato para tratamento com um composto ou um sal, prodroga ou isômero do mesmo, composto ou um sal, prodroga ou isômero do mesmo, e método.
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
HK1224186A1 (zh) 2017-08-18
EP3071198A1 (en) 2016-09-28
MX2016006583A (es) 2016-09-06
US20150139987A1 (en) 2015-05-21
AU2014353246A1 (en) 2016-06-16
EA201691039A1 (ru) 2016-11-30
PH12016500886A1 (en) 2016-06-20
CA2930069A1 (en) 2015-05-28
KR20160079124A (ko) 2016-07-05
JP2017505285A (ja) 2017-02-16
CN105764498A (zh) 2016-07-13
WO2015077154A1 (en) 2015-05-28

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