WO2004058268A3 - Verwendung von tetrahydrobiopterinderivaten zur behandlung und ernährung von patienten mit aminosäurestoffwechselstörungen - Google Patents

Verwendung von tetrahydrobiopterinderivaten zur behandlung und ernährung von patienten mit aminosäurestoffwechselstörungen Download PDF

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Publication number
WO2004058268A3
WO2004058268A3 PCT/EP2003/014262 EP0314262W WO2004058268A3 WO 2004058268 A3 WO2004058268 A3 WO 2004058268A3 EP 0314262 W EP0314262 W EP 0314262W WO 2004058268 A3 WO2004058268 A3 WO 2004058268A3
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WO
WIPO (PCT)
Prior art keywords
tetrahydrobiopterine
derivatives
patients
amino acid
treatment
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.)
Ceased
Application number
PCT/EP2003/014262
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English (en)
French (fr)
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WO2004058268A2 (de
Inventor
Ania Muntau-Heger
Adelbert A Roscher
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Biocrates Life Sciences AG
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Biocrates Life Sciences AG
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 Biocrates Life Sciences AG filed Critical Biocrates Life Sciences AG
Priority to AU2003294856A priority Critical patent/AU2003294856A1/en
Priority to EP03785822A priority patent/EP1575593A2/de
Priority to US10/539,842 priority patent/US20060211701A1/en
Publication of WO2004058268A2 publication Critical patent/WO2004058268A2/de
Publication of WO2004058268A3 publication Critical patent/WO2004058268A3/de
Anticipated expiration legal-status Critical
Priority to US15/211,180 priority patent/US20170042899A1/en
Ceased legal-status Critical Current

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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
    • A61K31/395—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
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A61K31/52—Purines, e.g. adenine
    • 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
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A—HUMAN NECESSITIES
    • A23—FOODS OR FOODSTUFFS; TREATMENT THEREOF, NOT COVERED BY OTHER CLASSES
    • A23L—FOODS, FOODSTUFFS OR NON-ALCOHOLIC BEVERAGES, NOT OTHERWISE PROVIDED FOR; PREPARATION OR TREATMENT THEREOF
    • A23L33/00—Modifying nutritive qualities of foods; Dietetic products; Preparation or treatment thereof
    • A23L33/10—Modifying nutritive qualities of foods; Dietetic products; Preparation or treatment thereof using additives
    • 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
    • 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
    • A61P3/02—Nutrients, e.g. vitamins, minerals
    • A—HUMAN NECESSITIES
    • A23—FOODS OR FOODSTUFFS; TREATMENT THEREOF, NOT COVERED BY OTHER CLASSES
    • A23V—INDEXING SCHEME RELATING TO FOODS, FOODSTUFFS OR NON-ALCOHOLIC BEVERAGES AND LACTIC OR PROPIONIC ACID BACTERIA USED IN FOODSTUFFS OR FOOD PREPARATION
    • A23V2002/00—Food compositions, function of food ingredients or processes for food or foodstuffs

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Nutrition Science (AREA)
  • Polymers & Plastics (AREA)
  • Mycology (AREA)
  • Food Science & Technology (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Coloring Foods And Improving Nutritive Qualities (AREA)

Abstract

Die vorliegende Erfindung betrifft die Verwendung von Tetrahydrobiopterin und seinen Derivaten zur Herstellung eines Medikamentes zur Verbesserung der Proteintoleranz zur Behandlung von Erkrankungen als Folge eines gestörten Aminosäurestoffwechsels, z.B. der Hyperphenylalaninämie. Die Erfindung betrifft ferner eine Zusammensetzung, welche Tetrahydrobiopterin oder Derivate davon sowie eine spezielle Aminosäuremischung enthält. Diese kann beispielsweise als phenylalaninarmes Spezialnahrungsmittel zur vollständigen Ernährung von hyperphenylalaninämishen Patienten eingesetzt werden. Bei den Untersuchungen im Rahmen der vorliegenden Erfindung hat sich herausgestellt, dass durch Tetrahydrobiopterin-Behandlung von Patienten, die Phenylalaninkonzentrationen über 200 µmol/l im Blut aufwiesen sich deren Phenylalaninkonzentrationen um 37 bis 92 % reduzierten.
PCT/EP2003/014262 2002-12-20 2003-12-15 Verwendung von tetrahydrobiopterinderivaten zur behandlung und ernährung von patienten mit aminosäurestoffwechselstörungen Ceased WO2004058268A2 (de)

Priority Applications (4)

Application Number Priority Date Filing Date Title
AU2003294856A AU2003294856A1 (en) 2002-12-20 2003-12-15 Use of von tetrahydrobiopterine derivatives in the treatment and nutrition of patients with amino acid metabolic disorders
EP03785822A EP1575593A2 (de) 2002-12-20 2003-12-15 VERWENDUNG VON TETRAHYDROBIOPTERINDERIVATEN ZUR BEHANDLUNG UND ERN HRUNG VON PATIENTEN MIT AMINOS URESTOFFWECHSELST&Ouml ;RUNGEN
US10/539,842 US20060211701A1 (en) 2002-12-20 2003-12-15 Use of von tetrahydrobiopterine derivatives in the treatment and nutrition of patients with amino acid metabolic disorders
US15/211,180 US20170042899A1 (en) 2002-12-20 2016-07-15 Use of Tetrahydrobiopterine Derivatives in the Treatment and Nutrition of Patients With Amino Acid Metabolic Disorders

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
DE10260263.8 2002-12-20
DE10260263A DE10260263A1 (de) 2002-12-20 2002-12-20 Verwendung von Tetrahydrobiopterinderivaten zur Behandlung und Ernährung von Patienten mit Aminosäurestoffwechselstörungen

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US10/539,842 A-371-Of-International US20060211701A1 (en) 2002-12-20 2003-12-15 Use of von tetrahydrobiopterine derivatives in the treatment and nutrition of patients with amino acid metabolic disorders
US15/211,180 Continuation US20170042899A1 (en) 2002-12-20 2016-07-15 Use of Tetrahydrobiopterine Derivatives in the Treatment and Nutrition of Patients With Amino Acid Metabolic Disorders

Publications (2)

Publication Number Publication Date
WO2004058268A2 WO2004058268A2 (de) 2004-07-15
WO2004058268A3 true WO2004058268A3 (de) 2004-09-30

Family

ID=32519249

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2003/014262 Ceased WO2004058268A2 (de) 2002-12-20 2003-12-15 Verwendung von tetrahydrobiopterinderivaten zur behandlung und ernährung von patienten mit aminosäurestoffwechselstörungen

Country Status (5)

Country Link
US (2) US20060211701A1 (de)
EP (1) EP1575593A2 (de)
AU (1) AU2003294856A1 (de)
DE (1) DE10260263A1 (de)
WO (1) WO2004058268A2 (de)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7566714B2 (en) 2003-11-17 2009-07-28 Biomarin Pharmaceutical Inc. Methods and compositions for the treatment of metabolic disorders
US9216178B2 (en) 2011-11-02 2015-12-22 Biomarin Pharmaceutical Inc. Dry blend formulation of tetrahydrobiopterin

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
WO2006055511A2 (en) * 2004-11-17 2006-05-26 Biomarin Pharmaceutical Inc. Stable tablet formulation of tetrahydrobiopterin
US20100009997A1 (en) * 2007-01-12 2010-01-14 Biomarin Pharmaceutical Inc. Method of treating a metabolic or neuropsychiarty disorder with a bh4 derivative prodrug
CA2675134A1 (en) * 2007-01-12 2008-07-24 Biomarin Pharmaceutical Inc. Pterin analogs
DE102007009650A1 (de) * 2007-02-26 2008-08-28 Beiersdorf Ag Kosmetisches Kombinationsprodukt zur Verbesserung des äußeren Erscheinungsbildes
WO2008128049A2 (en) * 2007-04-11 2008-10-23 Biomarin Pharmaceutical Inc. Methods of administering tetrahydrobiopterin, associated compositions, and methods of measuring
AU2008347005B2 (en) * 2008-01-03 2013-10-03 Biomarin Pharmaceutical Inc. Pterin analog for treating BH4 responsive condition
JP2011508781A (ja) * 2008-01-07 2011-03-17 ビオマリン プハルマセウトイカル インコーポレイテッド テトラヒドロビオプテリンの合成方法
US20110144117A1 (en) * 2008-08-12 2011-06-16 Orpha Swiss Gmbh Pharmaceutical Dosage Form Containing Tetrahydrobiopterin
JP6809781B2 (ja) * 2015-11-18 2021-01-06 白鳥製薬株式会社 プテリン誘導体又はその塩

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4778794A (en) * 1985-06-04 1988-10-18 Suntory Limited Pharmaceutical composition for the treatment of infantile autism
WO1995013803A1 (en) * 1993-11-15 1995-05-26 Cornell Research Foundation, Inc. Blocking induction of tetrahydrobiopterin to block induction of nitric oxide synthesis
US20020052374A1 (en) * 2000-06-07 2002-05-02 Rabelink Ton J. Pharmaceutical preparation containing at least folic acid or a folate and tetrahydrobiopterin (bh4) or derivatives thereof used for treating or preventing cardiovascular or neurological disorders by modulating of the activity of nitric oxide synthase (nos)

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JPS5925323A (ja) * 1982-03-03 1984-02-09 鐘淵化学工業株式会社 プテリン誘導体からなる脳内神経伝達物質の関わる疾病の治療剤
JPS5976086A (ja) * 1982-09-20 1984-04-28 ザ　ウエルカム　フアウンデ−シヨン　リミテツド プテリジン化合物
US4587340A (en) * 1983-09-19 1986-05-06 Burroughs Wellcome Co. Biopterin analogs
US4550109A (en) * 1984-05-31 1985-10-29 The Board Of Regents, The University Of Texas System Lipoidal biopterin compounds
DE3437944A1 (de) * 1984-10-17 1986-07-31 Biotest-Serum-Institut Gmbh, 6000 Frankfurt Verwendung von pterinen zur steigerung der aktivitaet von lymphokinen und anderen blutfaktoren, sowie ein diagnostisches oder therapeutisches praeparat, das pterine in kombination mit lymphokinen enthaelt
TW282398B (de) * 1993-12-22 1996-08-01 Bristol Myers Squibb Co
DE4418097A1 (de) * 1994-05-24 1995-11-30 Cassella Ag Verwendung von Tetrahydropteridin-Derivaten als Hemmstoffe der NO-Synthase
ATE218345T1 (de) * 1994-08-05 2002-06-15 Suntory Ltd Arzneimittel gegen spinocerebellare degeneration
US6428990B1 (en) * 1997-04-11 2002-08-06 Abbott Laboratories Human desaturase gene and uses thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4778794A (en) * 1985-06-04 1988-10-18 Suntory Limited Pharmaceutical composition for the treatment of infantile autism
WO1995013803A1 (en) * 1993-11-15 1995-05-26 Cornell Research Foundation, Inc. Blocking induction of tetrahydrobiopterin to block induction of nitric oxide synthesis
US20020052374A1 (en) * 2000-06-07 2002-05-02 Rabelink Ton J. Pharmaceutical preparation containing at least folic acid or a folate and tetrahydrobiopterin (bh4) or derivatives thereof used for treating or preventing cardiovascular or neurological disorders by modulating of the activity of nitric oxide synthase (nos)

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
DISSING I C ET AL: "TETRAHYDROBIOPTERIN AND PARKINSON'S DISEASE", ACTA NEUROLOGICA SCANDINAVICA, vol. 79, no. 6, 1989, pages 493 - 499, XP008029335, ISSN: 0001-6314 *
FURUKAWA, Y. ET AL: "Striatal biopterin and tyrosine hydroxylase protein reduction in dopa-responsive dystonia", NEUROLOGY (1999), 53(5), 1032-1041, XP008029137 *
HEITZER: "tetrahydrobiopterin improves...", DIABETOLOGIA, vol. 43, pages 1435 - 1438, XP001189711 *
JAPANESE COLLABORATIVE STUDY GROUP OF TETRAHYDROBIOPTERIN-RESPONSIVE PHENYLALANINE HYDROXYLASE DEFIC: "A nation-wide study of tetrahydrobiopterin-responsive phenylalanine hydroxylase deficiency in Japan", JOURNAL OF INHERITED METABOLIC DISEASE, vol. 24, no. Supplement 1, July 2001 (2001-07-01), 39th Annual Symposium of the Society for the Study of Inborn Errors of Metabolism;Prague, Czech Republic; September 04-07, 2001, pages 28, XP008029344, ISSN: 0141-8955 *
MILLER, L. ET AL: "Role of hydroxylase cofactor in regulating dopamine synthesis: relevance to neurological disorders", DOPAMINERGIC SYST. THEIR REGUL., [PROC. SYMP.] (1986), MEETING DATE 1984, 471-3. EDITOR(S): WOODRUFF, GEOFFREY N.;POAT, J. A.; ROBERTS, PETER J. PUBLISHER: VCH, WEINHEIM, FED. REP. GER., XP008029132 *
TANAKA Y ET AL: "On-off phenomenon in a child with tetrahydrobiopterin deficiency due to 6-pyruvoyl tetrahydropterin synthase deficiency (BH4 deficiency)", EUROPEAN JOURNAL OF PEDIATRICS 1989 GERMANY, vol. 148, no. 5, 1989, pages 450 - 452, XP008029347, ISSN: 0340-6199 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7566714B2 (en) 2003-11-17 2009-07-28 Biomarin Pharmaceutical Inc. Methods and compositions for the treatment of metabolic disorders
US9216178B2 (en) 2011-11-02 2015-12-22 Biomarin Pharmaceutical Inc. Dry blend formulation of tetrahydrobiopterin

Also Published As

Publication number Publication date
WO2004058268A2 (de) 2004-07-15
EP1575593A2 (de) 2005-09-21
AU2003294856A8 (en) 2004-07-22
AU2003294856A1 (en) 2004-07-22
US20060211701A1 (en) 2006-09-21
DE10260263A1 (de) 2004-07-15
US20170042899A1 (en) 2017-02-16

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