PL3923943T3 - Zależna od genotypu i dawki odpowiedź na asbti u pacjentów z niedoborem pompy eksportu soli żółciowych - Google Patents

Zależna od genotypu i dawki odpowiedź na asbti u pacjentów z niedoborem pompy eksportu soli żółciowych

Info

Publication number
PL3923943T3
PL3923943T3 PL20755296.9T PL20755296T PL3923943T3 PL 3923943 T3 PL3923943 T3 PL 3923943T3 PL 20755296 T PL20755296 T PL 20755296T PL 3923943 T3 PL3923943 T3 PL 3923943T3
Authority
PL
Poland
Prior art keywords
asbti
genotype
patients
dose
bile salt
Prior art date
Application number
PL20755296.9T
Other languages
English (en)
Inventor
Thomas JAECKLIN
Alejandro Dorenbaum
Original Assignee
Mirum 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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=72044264&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PL3923943(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Mirum Pharmaceuticals, Inc. filed Critical Mirum Pharmaceuticals, Inc.
Publication of PL3923943T3 publication Critical patent/PL3923943T3/pl

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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/4995—Pyrazines or piperazines forming part of bridged ring systems
    • 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
    • A61K31/55—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
    • A61K31/554—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole having at least one nitrogen and one sulfur as ring hetero atoms, e.g. clothiapine, diltiazem
    • 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
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/05—Dipeptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/10—Peptides having 12 to 20 amino acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/04—Antipruritics
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/106—Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/156—Polymorphic or mutational markers
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/08—Hepato-biliairy disorders other than hepatitis
    • G01N2800/085—Liver diseases, e.g. portal hypertension, fibrosis, cirrhosis, bilirubin

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Dermatology (AREA)
  • Analytical Chemistry (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Pathology (AREA)
  • Plant Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • Food Science & Technology (AREA)
  • Cell Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PL20755296.9T 2019-02-12 2020-02-12 Zależna od genotypu i dawki odpowiedź na asbti u pacjentów z niedoborem pompy eksportu soli żółciowych PL3923943T3 (pl)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962804523P 2019-02-12 2019-02-12
US201962863904P 2019-06-20 2019-06-20
US201962908431P 2019-09-30 2019-09-30
US201962932015P 2019-11-07 2019-11-07
PCT/US2020/017951 WO2020167964A1 (en) 2019-02-12 2020-02-12 Genotype and dose-dependent response to an asbti in patients with bile salt export pump deficiency

Publications (1)

Publication Number Publication Date
PL3923943T3 true PL3923943T3 (pl) 2024-12-09

Family

ID=72044264

Family Applications (2)

Application Number Title Priority Date Filing Date
PL20756251.3T PL3923970T3 (pl) 2019-02-12 2020-02-12 Sposoby zwiększania wzrostu u osobników pediatrycznych z cholestatyczną chorobą wątroby
PL20755296.9T PL3923943T3 (pl) 2019-02-12 2020-02-12 Zależna od genotypu i dawki odpowiedź na asbti u pacjentów z niedoborem pompy eksportu soli żółciowych

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PL20756251.3T PL3923970T3 (pl) 2019-02-12 2020-02-12 Sposoby zwiększania wzrostu u osobników pediatrycznych z cholestatyczną chorobą wątroby

Country Status (17)

Country Link
US (10) US20220152022A1 (pl)
EP (7) EP4464376A3 (pl)
JP (6) JP2022520121A (pl)
KR (3) KR20210137466A (pl)
CN (3) CN114096268A (pl)
AU (3) AU2020221834B2 (pl)
BR (3) BR112021015799A2 (pl)
CA (3) CA3129735A1 (pl)
DK (1) DK3923943T3 (pl)
ES (2) ES2982943T3 (pl)
FI (1) FI3923943T3 (pl)
HR (2) HRP20240801T1 (pl)
IL (3) IL285495A (pl)
MX (4) MX2021009623A (pl)
PL (2) PL3923970T3 (pl)
PT (1) PT3923943T (pl)
WO (3) WO2020167958A1 (pl)

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EP3400944B1 (en) 2010-11-04 2020-07-15 Albireo AB Ibat inhibitors for the treatment of liver diseases
JP6751020B2 (ja) 2014-06-25 2020-09-02 Ｅａファーマ株式会社 固形製剤及びその着色防止又は着色低減方法
US11801226B2 (en) 2018-06-20 2023-10-31 Albireo Ab Pharmaceutical formulation of odevixibat
SG11202012170PA (en) 2018-06-20 2021-01-28 Albireo Ab Pharmaceutical formulation of odevixibat
US10975045B2 (en) 2019-02-06 2021-04-13 Aibireo AB Benzothiazepine compounds and their use as bile acid modulators
US10941127B2 (en) 2019-02-06 2021-03-09 Albireo Ab Benzothiadiazepine compounds and their use as bile acid modulators
EP3921028B1 (en) 2019-02-06 2022-11-09 Albireo AB Benzothiadiazepine compounds and their use as bile acid modulators
ES2982943T3 (es) * 2019-02-12 2024-10-21 Mirum Pharmaceuticals Inc Métodos para aumentar el crecimiento en sujetos pediátricos con hepatopatía colestásica
PE20230234A1 (es) 2019-12-04 2023-02-07 Albireo Ab Compuestos de benzoti(di)azepina y su uso como moduladores del acido biliar
US11014898B1 (en) 2020-12-04 2021-05-25 Albireo Ab Benzothiazepine compounds and their use as bile acid modulators
JP7696898B2 (ja) 2019-12-04 2025-06-23 アルビレオ・アクチボラグ ベンゾチア（ジ）アゼピン化合物及び胆汁酸モジュレータとしてのそれらの使用
JP7748451B2 (ja) 2020-08-03 2025-10-02 アルビレオ・アクチボラグ ベンゾチア（ジ）アゼピン化合物及び胆汁酸モジュレータとしてのそれらの使用
EP4243831A1 (en) 2020-11-12 2023-09-20 Albireo AB Odevixibat for treating progressive familial intrahepatic cholestasis (pfic)
CN116710100A (zh) * 2020-11-12 2023-09-05 阿尔比里奥公司 用于治疗进行性家族性肝内胆汁淤积(pfic)的奥德昔巴特
WO2022101379A1 (en) 2020-11-12 2022-05-19 Albireo Ab Odevixibat for treating progressive familial intrahepatic cholestasis (pfic)
CA3198216A1 (en) 2020-12-04 2022-06-09 Albireo Ab Benzothia(di)azepine compounds and their use as bile acid modulators
TW202313579A (zh) 2021-06-03 2023-04-01 瑞典商艾爾比瑞歐公司 苯并噻(二)氮呯(benzothia(di)azepine)化合物及其作為膽酸調節劑之用途
TW202333722A (zh) * 2021-10-26 2023-09-01 美商米魯姆製藥公司 頂端鈉依賴型轉運蛋白抑制劑組合物
TW202333723A (zh) * 2021-10-26 2023-09-01 美商米魯姆製藥公司 頂端鈉依賴型膽酸轉運蛋白抑制劑(asbti)之給藥方法
TW202333724A (zh) * 2021-11-05 2023-09-01 美商米魯姆製藥公司 以迴腸膽酸轉運蛋白(ibat)抑制劑治療以增加無事件存活期(efs)
CN119053330A (zh) * 2022-04-22 2024-11-29 阿尔比里奥公司 皮下给药asbt抑制剂
CN120091821A (zh) * 2022-10-23 2025-06-03 米鲁姆制药公司 治疗进行性家族性肝内胆汁淤积症的方法
EP4611759A1 (en) 2022-11-03 2025-09-10 Albireo AB Treating alagille syndrome (algs)
EP4629997A1 (en) * 2022-12-09 2025-10-15 Albireo AB Asbt inhibitors in the treatment of renal diseases
US12296050B2 (en) * 2023-10-05 2025-05-13 Mirum Pharmaceuticals, Inc. Pharmaceutical compositions comprising maralixibat and uses thereof
WO2025093760A1 (en) 2023-11-03 2025-05-08 Albireo Ab Treating pfic2 with odevixibat

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