DK2613798T4 - Anvendelse af lysosomal sur lipase til behandling af patienter med mangel på lysosomal sur lipase - Google Patents
Anvendelse af lysosomal sur lipase til behandling af patienter med mangel på lysosomal sur lipase Download PDFInfo
- Publication number
- DK2613798T4 DK2613798T4 DK11758644.6T DK11758644T DK2613798T4 DK 2613798 T4 DK2613798 T4 DK 2613798T4 DK 11758644 T DK11758644 T DK 11758644T DK 2613798 T4 DK2613798 T4 DK 2613798T4
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- DK
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- Prior art keywords
- lal
- patient
- infusion
- administration
- exogenous
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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- 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
- A61K31/13—Amines
- A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
-
- 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
- A61K31/13—Amines
- A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
- A61K31/138—Aryloxyalkylamines, e.g. propranolol, tamoxifen, phenoxybenzamine
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- 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
- A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
- A61K31/365—Lactones
- A61K31/366—Lactones having six-membered rings, e.g. delta-lactones
-
- 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
- A61K31/397—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having four-membered rings, e.g. azetidine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
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- 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
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- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/18—Carboxylic ester hydrolases (3.1.1)
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/18—Carboxylic ester hydrolases (3.1.1)
- C12N9/20—Triglyceride splitting, e.g. by means of lipase
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/01—Carboxylic ester hydrolases (3.1.1)
- C12Y301/01013—Sterol esterase (3.1.1.13)
-
- 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
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Genetics & Genomics (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Investigating Or Analysing Biological Materials (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Claims (13)
- 21. Rekombinant human LAL til anvendelse ifølge krav 20, hvor infusionen er for én til fire timer.
- 22. Rekombinant human LAL til anvendelse ifølge krav 1, hvor indgivelsen er tilstrækkelig til at reducere lymfadenopati.
- 23. Rekombinant human LAL til anvendelse ifølge krav 1, hvor den humane patient er under 1 år, og indgivelsen er tilstrækkelig til at forhøje den humane patients væksthastighed.
- 24. Rekombinant human LAL til anvendelse ifølge krav 1, yderligere omfattende indgivelse af et andet terapeutikum.
- 25. Rekombinant human LAL til anvendelse ifølge krav 24, hvor det andet terapeutikum er et kolesterolsænkende lægemiddel.
- 26. Rekombinant human LAL til anvendelse ifølge krav 25, hvor det kolesterolsænkende lægemiddel er et statin.
- 27. Rekombinant human LAL til anvendelse ifølge krav 25, hvor det kolesterolsænkende lægemiddel er ezetimib.
- 28. Rekombinant human LAL til anvendelse ifølge krav 24, hvor det andet terapeutikum er et immunsuppressivum.
- 29. Rekombinant human LAL til anvendelse ifølge krav 24, hvor det andet terapeutikum er et antihistamin.
- 30. Rekombinant human LAL til anvendelse ifølge krav 29, hvor antihistaminet er diphenhydramin.
- 31. Rekombinant human LAL til anvendelse ifølge krav 30, hvor antihistaminet indgives, før LAL indgives, i en dosis på 1 mg til 5 mg pr kg legemsvægt af den humane patient.
- 32. Rekombinant human LAL til anvendelse ifølge krav 30, hvor antihistaminet indgives mellem 20 og 90 minutter før indgivelse af rekombinant human LAL.
- 33. Anvendelse af en rekombinant human lysosomal sur lipase (LAL) til fremstilling af et medikament til behandling af en human patient, der lider af LAL-mangel, ved at normalisere serumniveauerne af en levertransaminase hos den humane patient og reducere leverskader hos den humane patient, hvor den rekombinante humane LAL er til indgivelse til den humane patient mellem én gang hver 7 dage og én gang hver 30 dage, som defineret i et hvilket som helst af kravene 1 til 32.
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US40301110P | 2010-09-09 | 2010-09-09 | |
| US45601410P | 2010-10-29 | 2010-10-29 | |
| US201161432372P | 2011-01-13 | 2011-01-13 | |
| PCT/US2011/033699 WO2011133960A2 (en) | 2010-04-23 | 2011-04-23 | Lysosomal storage disease enzyme |
| PCT/US2011/051096 WO2012050695A1 (en) | 2010-09-09 | 2011-09-09 | Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| DK2613798T3 DK2613798T3 (da) | 2015-04-20 |
| DK2613798T4 true DK2613798T4 (da) | 2018-04-16 |
Family
ID=45806910
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK14200334.2T DK2977057T3 (da) | 2010-09-09 | 2011-09-09 | Isoleret rekombinant human N-glycosyleret lysosomal syrelipase |
| DK11758644.6T DK2613798T4 (da) | 2010-09-09 | 2011-09-09 | Anvendelse af lysosomal sur lipase til behandling af patienter med mangel på lysosomal sur lipase |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK14200334.2T DK2977057T3 (da) | 2010-09-09 | 2011-09-09 | Isoleret rekombinant human N-glycosyleret lysosomal syrelipase |
Country Status (30)
| Country | Link |
|---|---|
| US (5) | US8663631B2 (da) |
| EP (3) | EP2613798B2 (da) |
| JP (3) | JP5693728B2 (da) |
| KR (1) | KR20150038636A (da) |
| CN (2) | CN103200958A (da) |
| AR (1) | AR082953A1 (da) |
| AU (1) | AU2011314293B2 (da) |
| BR (2) | BR122019021757B1 (da) |
| CA (2) | CA3209456A1 (da) |
| CL (1) | CL2013000664A1 (da) |
| CO (1) | CO6710913A2 (da) |
| CY (1) | CY1116544T1 (da) |
| DK (2) | DK2977057T3 (da) |
| ES (2) | ES2535605T5 (da) |
| HK (2) | HK1220907A1 (da) |
| HR (1) | HRP20150438T1 (da) |
| HU (1) | HUE048688T2 (da) |
| IL (2) | IL225095A (da) |
| ME (1) | ME02062B (da) |
| MX (2) | MX2013002704A (da) |
| NZ (3) | NZ608292A (da) |
| PL (2) | PL2613798T5 (da) |
| PT (2) | PT2977057T (da) |
| RS (1) | RS53947B1 (da) |
| RU (1) | RU2550961C2 (da) |
| SG (2) | SG10201507199UA (da) |
| SI (2) | SI2977057T1 (da) |
| SM (1) | SMT201500108B (da) |
| TW (1) | TWI482625B (da) |
| WO (1) | WO2012050695A1 (da) |
Families Citing this family (14)
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| WO2001056596A1 (en) * | 2000-02-04 | 2001-08-09 | Children's Hospital Research Foundation | Use of lysosomal acid lipase for treating atherosclerosis and related diseases |
| WO2007030375A2 (en) * | 2005-09-08 | 2007-03-15 | Children's Hospital Medical Center | Lysosomal acid lipase therapy for nafld and related diseases |
| CN105214076B (zh) | 2010-04-23 | 2020-03-31 | 辛那杰瓦生物制药股份有限公司 | 溶酶体贮积病酶 |
| MX2013002704A (es) | 2010-09-09 | 2013-09-13 | Synageva Biopharma Corp | Uso de acido lipasa lisosomal para tratar la deficiencia de acido lipasa lisosomal en pacientes. |
| EP2675472A4 (en) | 2011-02-15 | 2014-09-17 | Synageva Biopharma Corp | METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE |
| EP2766477B1 (en) | 2011-10-12 | 2018-09-26 | Alexion Pharmaceuticals, Inc. | Recombinant human naglu protein and uses thereof |
| EP2820032A4 (en) * | 2012-03-02 | 2015-11-25 | Synageva Biopharma Corp | TRUNCATED LYSOSOMAL ACID LIPASE |
| CN106885910B (zh) * | 2015-12-15 | 2018-12-21 | 上海吉涛生物科技有限公司 | 肿瘤易感微环境检测装置及试剂盒 |
| WO2017218926A1 (en) | 2016-06-17 | 2017-12-21 | Alexion Pharmaceuticals, Inc. | Lysosomal acid lipase deficiency compositions and methods |
| WO2018039163A1 (en) | 2016-08-23 | 2018-03-01 | Alexion Pharmaceuticals, Inc. | Method of purifying a heterologous protein from an egg white |
| US10633705B2 (en) | 2016-10-14 | 2020-04-28 | Alexion Pharmaceuticals, Inc. | N-acetyl-alpha-D-glucosaminidase deficiency compositions and methods |
| US10738338B2 (en) | 2016-10-18 | 2020-08-11 | The Research Foundation for the State University | Method and composition for biocatalytic protein-oligonucleotide conjugation and protein-oligonucleotide conjugate |
| US11396499B2 (en) | 2018-12-12 | 2022-07-26 | University Of Washington | Lysosomal acid lipase assay |
| WO2021117122A1 (ja) * | 2019-12-10 | 2021-06-17 | 株式会社リボルナバイオサイエンス | ライソゾーム酸性リパーゼ欠損症の予防または治療剤 |
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