HRP20191710T1 - Postupci za liječenje poremećaja ciklusa uree - Google Patents
Postupci za liječenje poremećaja ciklusa uree Download PDFInfo
- Publication number
- HRP20191710T1 HRP20191710T1 HRP20191710T HRP20191710T1 HR P20191710 T1 HRP20191710 T1 HR P20191710T1 HR P20191710 T HRP20191710 T HR P20191710T HR P20191710 T1 HRP20191710 T1 HR P20191710T1
- Authority
- HR
- Croatia
- Prior art keywords
- phenylbutyrate
- dose
- bsa
- day
- glyceryl tri
- Prior art date
Links
- 238000000034 method Methods 0.000 title claims 7
- 208000028547 Inborn Urea Cycle disease Diseases 0.000 title claims 4
- 208000030954 urea cycle disease Diseases 0.000 title claims 4
- ZSDBFLMJVAGKOU-UHFFFAOYSA-N glycerol phenylbutyrate Chemical compound C=1C=CC=CC=1CCCC(=O)OCC(OC(=O)CCCC=1C=CC=CC=1)COC(=O)CCCC1=CC=CC=C1 ZSDBFLMJVAGKOU-UHFFFAOYSA-N 0.000 claims 8
- IJGRMHOSHXDMSA-UHFFFAOYSA-N Atomic nitrogen Chemical compound N#N IJGRMHOSHXDMSA-UHFFFAOYSA-N 0.000 claims 2
- 229940002612 prodrug Drugs 0.000 claims 2
- 239000000651 prodrug Substances 0.000 claims 2
- OBKXEAXTFZPCHS-UHFFFAOYSA-N 4-phenylbutyric acid Chemical compound OC(=O)CCCC1=CC=CC=C1 OBKXEAXTFZPCHS-UHFFFAOYSA-N 0.000 claims 1
- 229940079593 drug Drugs 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 229910052757 nitrogen Inorganic materials 0.000 claims 1
- 229950009215 phenylbutanoic acid Drugs 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- 230000002000 scavenging effect Effects 0.000 claims 1
- 229960002232 sodium phenylbutyrate Drugs 0.000 claims 1
- VPZRWNZGLKXFOE-UHFFFAOYSA-M sodium phenylbutyrate Chemical compound [Na+].[O-]C(=O)CCCC1=CC=CC=C1 VPZRWNZGLKXFOE-UHFFFAOYSA-M 0.000 claims 1
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
- A61K31/21—Esters, e.g. nitroglycerine, selenocyanates
- A61K31/215—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids
- A61K31/235—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids having an aromatic ring attached to a carboxyl group
-
- 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
-
- 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
-
- 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
- G01N33/6803—General methods of protein analysis not limited to specific proteins or families of proteins
- G01N33/6806—Determination of free amino acids
- G01N33/6812—Assays for specific amino acids
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2800/00—Detection or diagnosis of diseases
- G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2800/00—Detection or diagnosis of diseases
- G01N2800/70—Mechanisms involved in disease identification
- G01N2800/7057—(Intracellular) signaling and trafficking pathways
- G01N2800/7066—Metabolic pathways
- G01N2800/708—Nitrogen metabolism, e.g. urea cycle
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Hematology (AREA)
- Immunology (AREA)
- Biomedical Technology (AREA)
- Urology & Nephrology (AREA)
- Physics & Mathematics (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- General Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Cell Biology (AREA)
- Biotechnology (AREA)
- Food Science & Technology (AREA)
- Analytical Chemistry (AREA)
- Biochemistry (AREA)
- Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Emergency Medicine (AREA)
- Biophysics (AREA)
- Bioinformatics & Computational Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Investigating Or Analysing Biological Materials (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Plant Substances (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
Claims (7)
1. Postupak za određivanje učinkovite doze gliceril tri-[4-fenilbutirat] za liječenje poremećaja ciklusa uree kod subjekta u dobi od 6 do 17 godina kojemu je to potrebno koji uključuje:
izračunavanje ukupne površine tijela (BSA) za subjekta; i
uspoređivanje BSA s unaprijed određenom graničnom vrijednošću koja iznosi 1.3 m2,
naznačen time što je učinkovita doza prva doza od 7.3 do 8.79 g/m2/dan ako je BSA na ili iznad prethodno određene granične vrijednosti ili druga doza od 9 do 9.9 g/m2/dan ako je BSA ispod prethodno određene granične vrijednosti.
2. Postupak prema zahtjevu 1, naznačen time što prva doza iznosi 8 g/m2/dan.
3. Postupak prema zahtjevu 1 ili zahtjevu 2, naznačen time što druga doza iznosi 9 do 9.5 g/m2/dan.
4. Gliceril tri-[4-fenilbutirat] za uporabu u postupku za liječenje poremećaja ciklusa uree (UCD) kod subjekta u dobi od 6 do 17 godina kojemu je to potrebno, naznačen time što postupak uključuje sljedeće korake:
izračunavanje ukupne površine tijela (BSA) za subjekta,
uspoređivanje BSA s unaprijed određenom graničnom vrijednošću koja iznosi 1.3 m2, i
primjenu prve doze od 7.3 do 8.79 g/m2/dan gliceril tri-[4-fenilbutirata] ako je BSA na ili iznad prethodno određene granične vrijednosti ili druge doze od 9 do 9.9 g/m2/dan gliceril tri-[4-fenilbutirata] ako je BSA ispod prethodno određene granične vrijednosti.
5. Gliceril tri-[4-fenilbutirat] za uporabu kao u patentnom zahtjevu 4, naznačen time što prva doza iznosi 8 g/m2/dan.
6. Gliceril tri-[4-fenilbutirat] za uporabu kao u patentnom zahtjevu 4 ili zahtjevu 5, naznačen time što druga doza iznosi 9 do 9.5 g/m2/dan.
7. Postupak prema bilo kojem od zahtjeva 1-3, ili gliceril tri-[4-fenilbutirat] za uporabu kao u bilo kojem od patentnih zahtjeva 4-6, naznačen time što subjekt nije prethodno liječen s predlijekom PAA ili drugim lijekom za uklanjanje dušika, pri čemu je predlijek PAA izabran iz skupine koja sadrži gliceril tri-[4-fenilbutirat] i fenilbutirat ili njihovu farmaceutski prihvatljivu sol uključujući natrijev fenilbutirat.
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361890827P | 2013-10-14 | 2013-10-14 | |
| US201462044168P | 2014-08-29 | 2014-08-29 | |
| PCT/US2014/060543 WO2015057747A1 (en) | 2013-10-14 | 2014-10-14 | Methods of treating urea cycle disorders |
| EP14854074.3A EP2986325B1 (en) | 2013-10-14 | 2014-10-14 | Methods of treating urea cycle disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20191710T1 true HRP20191710T1 (hr) | 2019-12-13 |
Family
ID=52810198
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20191710 HRP20191710T1 (hr) | 2013-10-14 | 2019-09-20 | Postupci za liječenje poremećaja ciklusa uree |
Country Status (14)
| Country | Link |
|---|---|
| US (7) | US20150105469A1 (hr) |
| EP (1) | EP2986325B1 (hr) |
| CY (1) | CY1122220T1 (hr) |
| DK (1) | DK2986325T3 (hr) |
| ES (1) | ES2746999T3 (hr) |
| HK (1) | HK1219656A1 (hr) |
| HR (1) | HRP20191710T1 (hr) |
| HU (1) | HUE045106T2 (hr) |
| LT (1) | LT2986325T (hr) |
| PL (1) | PL2986325T3 (hr) |
| PT (1) | PT2986325T (hr) |
| RS (1) | RS59429B1 (hr) |
| SI (1) | SI2986325T1 (hr) |
| WO (1) | WO2015057747A1 (hr) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2760479T3 (en) | 2011-09-30 | 2017-07-17 | Horizon Therapeutics Llc | Nitrogen-removing drug for use in a method of treating a nitrogen retention disorder |
| LT2922576T (lt) | 2012-11-21 | 2018-02-12 | Horizon Therapeutics, Llc | Azotą surišančių vaistų, skirtų kepenų encefalopatijos gydymui, skyrimo ir įvertinimo būdai |
| WO2017147193A1 (en) * | 2016-02-22 | 2017-08-31 | Horizon Therapeutics, Llc | Treatment of urea cycle disorders in neonates and infants |
| US9914692B2 (en) | 2016-05-25 | 2018-03-13 | Horizon Therapeutics, Llc | Procedure for the preparation of 4-phenyl butyrate and uses thereof |
| US10668040B2 (en) | 2017-09-11 | 2020-06-02 | Horizon Therapeutics, Llc | Treatment of urea cycle disorders in neonates and infants |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE69612755T2 (de) | 1995-02-07 | 2001-08-23 | Brusilow Entpr Llc | Triglyceride und ethylester von phenylalkansäuren und phenylalkensäuren zur behandlung verschiedener erkrankungen |
| CA2735218A1 (en) | 2008-04-29 | 2009-11-05 | Ucyclyd Pharma, Inc. | Methods of treatment using ammonia-scavenging drugs |
| CA2735234A1 (en) * | 2008-08-29 | 2010-03-04 | Ucyclyd Pharma, Inc. | Dosing and monitoring patients on nitrogen-scavenging drugs |
| US9078865B2 (en) | 2009-07-24 | 2015-07-14 | Baylor College Of Medicine | Methods of modulation of branched chain acids and uses thereof |
| DK2760479T3 (en) | 2011-09-30 | 2017-07-17 | Horizon Therapeutics Llc | Nitrogen-removing drug for use in a method of treating a nitrogen retention disorder |
| WO2013158145A1 (en) | 2012-04-20 | 2013-10-24 | Bruce Scharschmidt | Methods of therapeutic monitoring of phenylacetic acid prodrugs |
-
2014
- 2014-10-14 RS RSP20191147 patent/RS59429B1/sr unknown
- 2014-10-14 SI SI201431340T patent/SI2986325T1/sl unknown
- 2014-10-14 PL PL14854074T patent/PL2986325T3/pl unknown
- 2014-10-14 HU HUE14854074A patent/HUE045106T2/hu unknown
- 2014-10-14 DK DK14854074.3T patent/DK2986325T3/da active
- 2014-10-14 EP EP14854074.3A patent/EP2986325B1/en active Active
- 2014-10-14 WO PCT/US2014/060543 patent/WO2015057747A1/en active Application Filing
- 2014-10-14 US US14/514,334 patent/US20150105469A1/en not_active Abandoned
- 2014-10-14 PT PT148540743T patent/PT2986325T/pt unknown
- 2014-10-14 LT LTEP14854074.3T patent/LT2986325T/lt unknown
- 2014-10-14 ES ES14854074T patent/ES2746999T3/es active Active
-
2015
- 2015-11-12 US US14/939,127 patent/US20160074356A1/en not_active Abandoned
-
2016
- 2016-07-01 HK HK16107680.2A patent/HK1219656A1/zh unknown
-
2017
- 2017-06-30 US US15/639,171 patent/US20170354631A1/en not_active Abandoned
- 2017-08-25 US US15/687,141 patent/US20170354632A1/en not_active Abandoned
- 2017-08-25 US US15/687,144 patent/US20180015065A1/en not_active Abandoned
- 2017-09-08 US US15/699,188 patent/US20180021292A1/en not_active Abandoned
-
2019
- 2019-09-16 CY CY20191100964T patent/CY1122220T1/el unknown
- 2019-09-20 HR HRP20191710 patent/HRP20191710T1/hr unknown
-
2020
- 2020-03-06 US US16/811,861 patent/US20200405678A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| US20150105469A1 (en) | 2015-04-16 |
| WO2015057747A1 (en) | 2015-04-23 |
| DK2986325T3 (da) | 2019-09-23 |
| EP2986325A1 (en) | 2016-02-24 |
| EP2986325B1 (en) | 2019-06-26 |
| US20180021292A1 (en) | 2018-01-25 |
| SI2986325T1 (sl) | 2020-02-28 |
| LT2986325T (lt) | 2019-10-10 |
| US20180015065A1 (en) | 2018-01-18 |
| PT2986325T (pt) | 2019-10-09 |
| PL2986325T3 (pl) | 2020-03-31 |
| RS59429B1 (sr) | 2019-11-29 |
| HUE045106T2 (hu) | 2019-12-30 |
| US20160074356A1 (en) | 2016-03-17 |
| HK1219656A1 (zh) | 2017-04-13 |
| EP2986325A4 (en) | 2016-07-27 |
| ES2746999T3 (es) | 2020-03-09 |
| US20200405678A1 (en) | 2020-12-31 |
| US20170354632A1 (en) | 2017-12-14 |
| US20170354631A1 (en) | 2017-12-14 |
| CY1122220T1 (el) | 2020-11-25 |
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