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

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/185Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19Carboxylic acids, e.g. valproic acid
    • A61K31/192Carboxylic acids, e.g. valproic acid having aromatic groups, e.g. sulindac, 2-aryl-propionic acids, ethacrynic acid 
    • 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • A61K31/4468Non condensed piperidines, e.g. piperocaine having a nitrogen directly attached in position 4, e.g. clebopride, fentanyl
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/395Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/40Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against enzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination 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)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Diabetes (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Endocrinology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Emergency 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
US201461974725P 2014-04-03 2014-04-03
US201461974785P 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 (2)
EP (1) EP3071198A1 (2)
JP (1) JP2017505285A (2)
KR (1) KR20160079124A (2)
CN (1) CN105764498A (2)
AU (1) AU2014353246A1 (2)
CA (1) CA2930069A1 (2)
CL (1) CL2016001215A1 (2)
EA (1) EA201691039A1 (2)
HK (1) HK1224186A1 (2)
IL (1) IL245748A0 (2)
MX (1) MX2016006583A (2)
PH (1) PH12016500886A1 (2)
WO (1) WO2015077154A1 (2)

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
US10125369B2 (en) 2012-12-05 2018-11-13 Alnylam Pharmaceuticals, Inc. 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
PT3119384T (pt) 2014-03-20 2018-12-12 Cymabay Therapeutics Inc Tratamento de doenças colestáticas intra-hepáticas
US10272058B2 (en) 2014-03-20 2019-04-30 Cymabay Therapeutics, Inc. Treatment of intrahepatic cholestatic diseases
BR112016023269A8 (pt) 2014-04-11 2021-06-29 Cymabay Therapeutics Inc uso de ácido (r)-2-(4-((2-etóxi-3-(4-(trifluormetil)fenóxi)propil)-tio)-2-metilfenóxi)-acético ou sal do mesmo na fabricação de um medicamento para tratar doença hepática gordurosa não alcoólica ou esteato-hepatite não alcoólica
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
CN118697894A (zh) * 2015-08-25 2024-09-27 阿尔尼拉姆医药品有限公司 用于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
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
IL293308B2 (en) * 2016-04-28 2024-12-01 Regeneron Pharma 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
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
US20220040332A1 (en) * 2018-12-20 2022-02-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
SG11202107614PA (en) 2019-01-18 2021-08-30 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
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
US20230285376A1 (en) 2020-07-29 2023-09-14 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
EP1667964B1 (en) 2003-09-19 2009-07-22 Janssen Pharmaceutica N.V. 4-((phenoxyalkyl)thio)-phenoxyacetic acids and analogs
US7932268B2 (en) 2004-03-05 2011-04-26 The Trustees Of The University Of 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
JP5694941B2 (ja) * 2008-10-17 2015-04-01 シーマベイ セラピューティクス,インコーポレイティド 小型高密度ldl粒子を減少させる方法
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
CN105764498A (zh) 2016-07-13
MX2016006583A (es) 2016-09-06
CA2930069A1 (en) 2015-05-28
AU2014353246A1 (en) 2016-06-16
WO2015077154A1 (en) 2015-05-28
US20150139987A1 (en) 2015-05-21
KR20160079124A (ko) 2016-07-05
EP3071198A1 (en) 2016-09-28
JP2017505285A (ja) 2017-02-16
PH12016500886A1 (en) 2016-06-20
HK1224186A1 (zh) 2017-08-18
EA201691039A1 (ru) 2016-11-30

Similar Documents

Publication Publication Date Title
CL2016001215A1 (es) Tratamiento de hipercolesterolemia familiar homocigota.
CL2015001442A1 (es) Metodo para tratar enfermedades pulmonares que comprende el uso de compuestos derivados del acido obeticolico.
HUE042134T2 (hu) Monobaktám szerves vegyületek bakteriális fertõzések kezelésére
EA201791420A1 (ru) Соединения триазолопиримидина и их применения
IL247452A0 (en) Compounds for treatment of complement mediated disorders
PH12016502382A1 (en) Substituted indazole compounds as irak4 inhibitors
EA201691625A1 (ru) Ароматические гетероциклические соединения как противовоспалительные соединения
CL2016001458A1 (es) Derivado de sulfonamida o sales de adición de ácido farmaceuticamente aceptables del mismo.
EA201591634A1 (ru) Дигидропиридопиримидиновые соединения
EP3030323A4 (en) Kdm1a inhibitors for the treatment of disease
UY34780A (es) Vectores virales para el tratamiento de distrofia retiniana
UY36314A (es) NUEVOS COMPUESTOS ANTAGONISTAS DE INTEGRINA avß6.
PL2852391T3 (pl) Układy do leczenia infekcji płuc
SI2953904T1 (sl) Postopek za nadzorovanje izdelave biocida
IL247334A0 (en) Inhibitors of leukotriene-mediated activity for treating side effects of statin therapy
UY34616A (es) Nuevo compuesto útil para el tratamiento de enfermedades degenerativas e inflamatorias.
HUE053443T2 (hu) Heteroaril vegyületek szembetegségek kezelésére
HRP20181570T1 (hr) Režim doziranja spoja fgf-18
CL2016003290A1 (es) Tratamiento de hipertrigliceridemia severa.
EA201591915A1 (ru) Соединения феноксиэтил дигидро-1h-изохинолина
HRP20181799T1 (hr) Optimizirana farmaceutska formula, namijenjena liječenju upalnih promjena jednjaka
MX2016015543A (es) Proceso para la preparacion de 5-fluoro-1h-pirazoles a partir de hexafluoropropeno.
HUE051295T2 (hu) Módosított fibroblaszt növekedési faktorok-1 szembetegségek kezelésére
EA201790687A1 (ru) Хинолинкарбоксамиды для применения в лечении множественной миеломы
EA201791099A1 (ru) Хинолин карбоксамиды для применения в лечении лейкоза