CA2947062A1 - Nitisinone dosing regimens for the treatment of alkaptonuria - Google Patents
Nitisinone dosing regimens for the treatment of alkaptonuria Download PDFInfo
- Publication number
- CA2947062A1 CA2947062A1 CA2947062A CA2947062A CA2947062A1 CA 2947062 A1 CA2947062 A1 CA 2947062A1 CA 2947062 A CA2947062 A CA 2947062A CA 2947062 A CA2947062 A CA 2947062A CA 2947062 A1 CA2947062 A1 CA 2947062A1
- Authority
- CA
- Canada
- Prior art keywords
- nitisinone
- dose
- administered
- hga
- tyrosine
- Prior art date
- Legal status (The legal status 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 status listed.)
- Abandoned
Links
- OUBCNLGXQFSTLU-UHFFFAOYSA-N nitisinone Chemical compound [O-][N+](=O)C1=CC(C(F)(F)F)=CC=C1C(=O)C1C(=O)CCCC1=O OUBCNLGXQFSTLU-UHFFFAOYSA-N 0.000 title claims abstract description 112
- 229960001721 nitisinone Drugs 0.000 title claims abstract description 112
- 238000011282 treatment Methods 0.000 title claims abstract description 30
- 206010001689 alkaptonuria Diseases 0.000 title claims abstract description 29
- 239000008194 pharmaceutical composition Substances 0.000 claims abstract description 11
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 5
- 239000000203 mixture Substances 0.000 claims description 3
- OUYCCCASQSFEME-QMMMGPOBSA-N L-tyrosine Chemical compound OC(=O)[C@@H](N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-QMMMGPOBSA-N 0.000 description 44
- OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Natural products OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 description 44
- 210000002966 serum Anatomy 0.000 description 39
- 230000036325 urinary excretion Effects 0.000 description 18
- 230000007423 decrease Effects 0.000 description 11
- 231100000673 dose–response relationship Toxicity 0.000 description 7
- 230000000694 effects Effects 0.000 description 6
- IGMNYECMUMZDDF-UHFFFAOYSA-N homogentisic acid Chemical compound OC(=O)CC1=CC(O)=CC=C1O IGMNYECMUMZDDF-UHFFFAOYSA-N 0.000 description 6
- 210000002700 urine Anatomy 0.000 description 6
- 108010068327 4-hydroxyphenylpyruvate dioxygenase Proteins 0.000 description 5
- COLNVLDHVKWLRT-QMMMGPOBSA-N L-phenylalanine Chemical compound OC(=O)[C@@H](N)CC1=CC=CC=C1 COLNVLDHVKWLRT-QMMMGPOBSA-N 0.000 description 5
- 238000000034 method Methods 0.000 description 5
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Natural products OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 description 5
- GACSIVHAIFQKTC-OWOJBTEDSA-N 4-fumarylacetoacetic acid Chemical compound OC(=O)CC(=O)CC(=O)\C=C\C(O)=O GACSIVHAIFQKTC-OWOJBTEDSA-N 0.000 description 4
- 102100028626 4-hydroxyphenylpyruvate dioxygenase Human genes 0.000 description 4
- GACSIVHAIFQKTC-UPHRSURJSA-N 4-maleylacetoacetic acid Chemical compound OC(=O)CC(=O)CC(=O)\C=C/C(O)=O GACSIVHAIFQKTC-UPHRSURJSA-N 0.000 description 4
- 102000030513 Homogentisate 1,2-Dioxygenase Human genes 0.000 description 4
- 108700023439 Homogentisate 1,2-dioxygenases Proteins 0.000 description 4
- 230000015572 biosynthetic process Effects 0.000 description 4
- 235000005911 diet Nutrition 0.000 description 4
- 238000001294 liquid chromatography-tandem mass spectrometry Methods 0.000 description 4
- KRKNYBCHXYNGOX-UHFFFAOYSA-N citric acid Chemical compound OC(=O)CC(O)(C(O)=O)CC(O)=O KRKNYBCHXYNGOX-UHFFFAOYSA-N 0.000 description 3
- 230000003247 decreasing effect Effects 0.000 description 3
- 230000037213 diet Effects 0.000 description 3
- 239000000049 pigment Substances 0.000 description 3
- KKADPXVIOXHVKN-UHFFFAOYSA-N 4-hydroxyphenylpyruvic acid Chemical compound OC(=O)C(=O)CC1=CC=C(O)C=C1 KKADPXVIOXHVKN-UHFFFAOYSA-N 0.000 description 2
- CIWBSHSKHKDKBQ-JLAZNSOCSA-N Ascorbic acid Chemical compound OC[C@H](O)[C@H]1OC(=O)C(O)=C1O CIWBSHSKHKDKBQ-JLAZNSOCSA-N 0.000 description 2
- RAPRJRLALQKSHB-UHFFFAOYSA-N benzoquinoneacetic acid Chemical compound OC(=O)CC1=CC(=O)C=CC1=O RAPRJRLALQKSHB-UHFFFAOYSA-N 0.000 description 2
- 208000010877 cognitive disease Diseases 0.000 description 2
- 230000007547 defect Effects 0.000 description 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 2
- 230000029142 excretion Effects 0.000 description 2
- 239000007788 liquid Substances 0.000 description 2
- 230000002503 metabolic effect Effects 0.000 description 2
- 230000036407 pain Effects 0.000 description 2
- 238000011002 quantification Methods 0.000 description 2
- 208000024891 symptom Diseases 0.000 description 2
- 201000011296 tyrosinemia Diseases 0.000 description 2
- 230000002485 urinary effect Effects 0.000 description 2
- WVXVAFQBNTVPCT-TYYBGVCCSA-N (e)-but-2-enedioic acid;3-oxobutanoic acid Chemical compound CC(=O)CC(O)=O.OC(=O)\C=C\C(O)=O WVXVAFQBNTVPCT-TYYBGVCCSA-N 0.000 description 1
- 208000007848 Alcoholism Diseases 0.000 description 1
- 208000024172 Cardiovascular disease Diseases 0.000 description 1
- 208000028698 Cognitive impairment Diseases 0.000 description 1
- 206010056476 Corneal irritation Diseases 0.000 description 1
- ZZZCUOFIHGPKAK-UHFFFAOYSA-N D-erythro-ascorbic acid Natural products OCC1OC(=O)C(O)=C1O ZZZCUOFIHGPKAK-UHFFFAOYSA-N 0.000 description 1
- 206010013654 Drug abuse Diseases 0.000 description 1
- 108060006698 EGF receptor Proteins 0.000 description 1
- LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 description 1
- 102100029115 Fumarylacetoacetase Human genes 0.000 description 1
- 208000010412 Glaucoma Diseases 0.000 description 1
- 102000001554 Hemoglobins Human genes 0.000 description 1
- 108010054147 Hemoglobins Proteins 0.000 description 1
- 208000028782 Hereditary disease Diseases 0.000 description 1
- 108090000604 Hydrolases Proteins 0.000 description 1
- 206010020751 Hypersensitivity Diseases 0.000 description 1
- 206010020772 Hypertension Diseases 0.000 description 1
- 208000000913 Kidney Calculi Diseases 0.000 description 1
- 208000024556 Mendelian disease Diseases 0.000 description 1
- 102000008109 Mixed Function Oxygenases Human genes 0.000 description 1
- 108010074633 Mixed Function Oxygenases Proteins 0.000 description 1
- 206010028980 Neoplasm Diseases 0.000 description 1
- 206010029148 Nephrolithiasis Diseases 0.000 description 1
- 208000012902 Nervous system disease Diseases 0.000 description 1
- 208000025966 Neurological disease Diseases 0.000 description 1
- 201000010394 Ochronosis Diseases 0.000 description 1
- ZLMJMSJWJFRBEC-UHFFFAOYSA-N Potassium Chemical compound [K] ZLMJMSJWJFRBEC-UHFFFAOYSA-N 0.000 description 1
- 208000035977 Rare disease Diseases 0.000 description 1
- QAOWNCQODCNURD-UHFFFAOYSA-N Sulfuric acid Chemical compound OS(O)(=O)=O QAOWNCQODCNURD-UHFFFAOYSA-N 0.000 description 1
- 102000016540 Tyrosine aminotransferases Human genes 0.000 description 1
- 108010042606 Tyrosine transaminase Proteins 0.000 description 1
- 208000032001 Tyrosinemia type 1 Diseases 0.000 description 1
- 229930003268 Vitamin C Natural products 0.000 description 1
- 238000009825 accumulation Methods 0.000 description 1
- 239000004480 active ingredient Substances 0.000 description 1
- 206010001584 alcohol abuse Diseases 0.000 description 1
- 208000025746 alcohol use disease Diseases 0.000 description 1
- 208000026935 allergic disease Diseases 0.000 description 1
- 230000007815 allergy Effects 0.000 description 1
- 150000001413 amino acids Chemical class 0.000 description 1
- 230000036592 analgesia Effects 0.000 description 1
- 230000002917 arthritic effect Effects 0.000 description 1
- 206010003246 arthritis Diseases 0.000 description 1
- 230000000903 blocking effect Effects 0.000 description 1
- 238000004820 blood count Methods 0.000 description 1
- 230000036772 blood pressure Effects 0.000 description 1
- 230000037396 body weight Effects 0.000 description 1
- 235000021152 breakfast Nutrition 0.000 description 1
- 239000000872 buffer Substances 0.000 description 1
- 201000011510 cancer Diseases 0.000 description 1
- 239000002775 capsule Substances 0.000 description 1
- 210000000845 cartilage Anatomy 0.000 description 1
- 230000015556 catabolic process Effects 0.000 description 1
- 238000006243 chemical reaction Methods 0.000 description 1
- 230000003920 cognitive function Effects 0.000 description 1
- 238000004891 communication Methods 0.000 description 1
- 230000002860 competitive effect Effects 0.000 description 1
- 210000002808 connective tissue Anatomy 0.000 description 1
- 239000000470 constituent Substances 0.000 description 1
- 239000013078 crystal Substances 0.000 description 1
- 230000006378 damage Effects 0.000 description 1
- 230000007812 deficiency Effects 0.000 description 1
- 238000006731 degradation reaction Methods 0.000 description 1
- 238000013461 design Methods 0.000 description 1
- 238000003745 diagnosis Methods 0.000 description 1
- 230000003205 diastolic effect Effects 0.000 description 1
- 230000000378 dietary effect Effects 0.000 description 1
- 235000020805 dietary restrictions Nutrition 0.000 description 1
- 201000010099 disease Diseases 0.000 description 1
- 208000035475 disorder Diseases 0.000 description 1
- 239000006185 dispersion Substances 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 230000007717 exclusion Effects 0.000 description 1
- 238000013213 extrapolation Methods 0.000 description 1
- 230000036541 health Effects 0.000 description 1
- 210000003709 heart valve Anatomy 0.000 description 1
- 229920006130 high-performance polyamide Polymers 0.000 description 1
- 230000001771 impaired effect Effects 0.000 description 1
- 239000003112 inhibitor Substances 0.000 description 1
- 230000005764 inhibitory process Effects 0.000 description 1
- 239000000543 intermediate Substances 0.000 description 1
- 206010023365 keratopathy Diseases 0.000 description 1
- 230000007774 longterm Effects 0.000 description 1
- 230000036210 malignancy Effects 0.000 description 1
- 238000004519 manufacturing process Methods 0.000 description 1
- 238000005259 measurement Methods 0.000 description 1
- 230000037353 metabolic pathway Effects 0.000 description 1
- 230000004060 metabolic process Effects 0.000 description 1
- 229940048333 nitisinone 2 mg Drugs 0.000 description 1
- 208000029347 ochronosis disease Diseases 0.000 description 1
- 229940100692 oral suspension Drugs 0.000 description 1
- 229940039219 orfadin Drugs 0.000 description 1
- 230000001590 oxidative effect Effects 0.000 description 1
- KXFJZKUFXHWWAJ-UHFFFAOYSA-N p-hydroxybenzoylformic acid Natural products OC(=O)C(=O)C1=CC=C(O)C=C1 KXFJZKUFXHWWAJ-UHFFFAOYSA-N 0.000 description 1
- 238000011458 pharmacological treatment Methods 0.000 description 1
- 238000000554 physical therapy Methods 0.000 description 1
- 229920000642 polymer Polymers 0.000 description 1
- 229910052700 potassium Inorganic materials 0.000 description 1
- 239000011591 potassium Substances 0.000 description 1
- 238000001556 precipitation Methods 0.000 description 1
- 238000002203 pretreatment Methods 0.000 description 1
- 230000008569 process Effects 0.000 description 1
- 230000000750 progressive effect Effects 0.000 description 1
- 210000002307 prostate Anatomy 0.000 description 1
- 208000020016 psychiatric disease Diseases 0.000 description 1
- 238000005070 sampling Methods 0.000 description 1
- 239000000243 solution Substances 0.000 description 1
- 208000011117 substance-related disease Diseases 0.000 description 1
- 235000011149 sulphuric acid Nutrition 0.000 description 1
- 238000001356 surgical procedure Methods 0.000 description 1
- 239000000725 suspension Substances 0.000 description 1
- 239000003826 tablet Substances 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
- 231100000331 toxic Toxicity 0.000 description 1
- 230000002588 toxic effect Effects 0.000 description 1
- 239000003053 toxin Substances 0.000 description 1
- 235000019154 vitamin C Nutrition 0.000 description 1
- 239000011718 vitamin C Substances 0.000 description 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
- A61K31/12—Ketones
- A61K31/122—Ketones having the oxygen directly attached to a ring, e.g. quinones, vitamin K1, anthralin
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Veterinary Medicine (AREA)
- Life Sciences & Earth Sciences (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nutrition Science (AREA)
- Physiology (AREA)
- Obesity (AREA)
- Rheumatology (AREA)
- Hematology (AREA)
- Immunology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Diabetes (AREA)
- Physical Education & Sports Medicine (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| SE1450521-8 | 2014-04-30 | ||
| SE1450521 | 2014-04-30 | ||
| PCT/EP2015/059352 WO2015165972A1 (en) | 2014-04-30 | 2015-04-29 | Nitisinone dosing regimens for the treatment of alkaptonuria |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2947062A1 true CA2947062A1 (en) | 2015-11-05 |
Family
ID=53175441
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2947062A Abandoned CA2947062A1 (en) | 2014-04-30 | 2015-04-29 | Nitisinone dosing regimens for the treatment of alkaptonuria |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20170049716A1 (enExample) |
| EP (1) | EP3137066A1 (enExample) |
| JP (1) | JP2017514820A (enExample) |
| AU (1) | AU2015254669A1 (enExample) |
| BR (1) | BR112016024756A2 (enExample) |
| CA (1) | CA2947062A1 (enExample) |
| IL (1) | IL248506A0 (enExample) |
| MA (1) | MA39918A (enExample) |
| SG (1) | SG11201608957VA (enExample) |
| TN (1) | TN2016000479A1 (enExample) |
| WO (1) | WO2015165972A1 (enExample) |
Family Cites Families (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2723320B1 (en) | 2011-06-23 | 2016-01-13 | Swedish Orphan Biovitrum International AB | Liquid pharmaceutical composition comprising nitisinone |
-
2015
- 2015-04-29 AU AU2015254669A patent/AU2015254669A1/en not_active Abandoned
- 2015-04-29 SG SG11201608957VA patent/SG11201608957VA/en unknown
- 2015-04-29 WO PCT/EP2015/059352 patent/WO2015165972A1/en not_active Ceased
- 2015-04-29 TN TN2016000479A patent/TN2016000479A1/en unknown
- 2015-04-29 BR BR112016024756A patent/BR112016024756A2/pt not_active IP Right Cessation
- 2015-04-29 JP JP2016564948A patent/JP2017514820A/ja active Pending
- 2015-04-29 MA MA039918A patent/MA39918A/fr unknown
- 2015-04-29 CA CA2947062A patent/CA2947062A1/en not_active Abandoned
- 2015-04-29 US US15/307,622 patent/US20170049716A1/en not_active Abandoned
- 2015-04-29 EP EP15722113.6A patent/EP3137066A1/en not_active Withdrawn
-
2016
- 2016-10-26 IL IL248506A patent/IL248506A0/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| WO2015165972A1 (en) | 2015-11-05 |
| IL248506A0 (en) | 2016-12-29 |
| AU2015254669A1 (en) | 2016-12-15 |
| JP2017514820A (ja) | 2017-06-08 |
| MA39918A (fr) | 2017-03-08 |
| TN2016000479A1 (en) | 2018-04-04 |
| US20170049716A1 (en) | 2017-02-23 |
| SG11201608957VA (en) | 2016-11-29 |
| BR112016024756A2 (pt) | 2017-08-15 |
| EP3137066A1 (en) | 2017-03-08 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FZDE | Discontinued |
Effective date: 20200831 |