WO2005049000A3 - Treatment of phenylketonurias with bh4 - Google Patents

Treatment of phenylketonurias with bh4 Download PDF

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Publication number
WO2005049000A3
WO2005049000A3 PCT/US2004/038296 US2004038296W WO2005049000A3 WO 2005049000 A3 WO2005049000 A3 WO 2005049000A3 US 2004038296 W US2004038296 W US 2004038296W WO 2005049000 A3 WO2005049000 A3 WO 2005049000A3
Authority
WO
WIPO (PCT)
Prior art keywords
compositions
phenylketonurias
treatment
hyperphenylalaninemia
contemplated
Prior art date
Application number
PCT/US2004/038296
Other languages
French (fr)
Other versions
WO2005049000A2 (en
Inventor
Dan Oppenheimer
Emil D Kakkis
Frederic D Price
Alejandro Dorenbaum
Original Assignee
Biomarin Pharm 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=34619513&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=WO2005049000(A3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority to MXPA06005451A priority Critical patent/MXPA06005451A/en
Priority to DK04819152.2T priority patent/DK1708690T3/en
Priority to ES04819152.2T priority patent/ES2596827T3/en
Priority to PL16178739T priority patent/PL3138566T3/en
Priority to BRPI0416566-7A priority patent/BRPI0416566A/en
Priority to EP04819152.2A priority patent/EP1708690B1/en
Priority to EP21209606.9A priority patent/EP3977999A1/en
Priority to AU2004291149A priority patent/AU2004291149C1/en
Priority to JP2006539992A priority patent/JP2007511536A/en
Priority to CA2545584A priority patent/CA2545584C/en
Priority to EP16178739.5A priority patent/EP3138566B1/en
Priority to CN2004800338351A priority patent/CN1905863B/en
Application filed by Biomarin Pharm Inc filed Critical Biomarin Pharm Inc
Publication of WO2005049000A2 publication Critical patent/WO2005049000A2/en
Publication of WO2005049000A3 publication Critical patent/WO2005049000A3/en
Priority to IL175529A priority patent/IL175529A/en
Priority to HK07102354.9A priority patent/HK1094875A1/en
Priority to CY20161101054T priority patent/CY1118171T1/en
Priority to CY20221100068T priority patent/CY1124931T1/en

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
    • A61K31/505—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 COVERED BY SUBCLASSES A21D OR A23B-A23J; THEIR PREPARATION OR TREATMENT, e.g. COOKING, MODIFICATION OF NUTRITIVE QUALITIES, PHYSICAL TREATMENT; PRESERVATION OF FOODS OR FOODSTUFFS, IN GENERAL
    • 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
    • A23L33/15—Vitamins
    • A—HUMAN NECESSITIES
    • A23—FOODS OR FOODSTUFFS; TREATMENT THEREOF, NOT COVERED BY OTHER CLASSES
    • A23L—FOODS, FOODSTUFFS, OR NON-ALCOHOLIC BEVERAGES, NOT COVERED BY SUBCLASSES A21D OR A23B-A23J; THEIR PREPARATION OR TREATMENT, e.g. COOKING, MODIFICATION OF NUTRITIVE QUALITIES, PHYSICAL TREATMENT; PRESERVATION OF FOODS OR FOODSTUFFS, IN GENERAL
    • 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
    • A23L33/15—Vitamins
    • A23L33/155—Vitamins A or D
    • A—HUMAN NECESSITIES
    • A23—FOODS OR FOODSTUFFS; TREATMENT THEREOF, NOT COVERED BY OTHER CLASSES
    • A23L—FOODS, FOODSTUFFS, OR NON-ALCOHOLIC BEVERAGES, NOT COVERED BY SUBCLASSES A21D OR A23B-A23J; THEIR PREPARATION OR TREATMENT, e.g. COOKING, MODIFICATION OF NUTRITIVE QUALITIES, PHYSICAL TREATMENT; PRESERVATION OF FOODS OR FOODSTUFFS, IN GENERAL
    • 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
    • A23L33/16—Inorganic salts, minerals or trace elements
    • A—HUMAN NECESSITIES
    • A23—FOODS OR FOODSTUFFS; TREATMENT THEREOF, NOT COVERED BY OTHER CLASSES
    • A23L—FOODS, FOODSTUFFS, OR NON-ALCOHOLIC BEVERAGES, NOT COVERED BY SUBCLASSES A21D OR A23B-A23J; THEIR PREPARATION OR TREATMENT, e.g. COOKING, MODIFICATION OF NUTRITIVE QUALITIES, PHYSICAL TREATMENT; PRESERVATION OF FOODS OR FOODSTUFFS, IN GENERAL
    • 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
    • A23L33/17—Amino acids, peptides or proteins
    • A—HUMAN NECESSITIES
    • A23—FOODS OR FOODSTUFFS; TREATMENT THEREOF, NOT COVERED BY OTHER CLASSES
    • A23L—FOODS, FOODSTUFFS, OR NON-ALCOHOLIC BEVERAGES, NOT COVERED BY SUBCLASSES A21D OR A23B-A23J; THEIR PREPARATION OR TREATMENT, e.g. COOKING, MODIFICATION OF NUTRITIVE QUALITIES, PHYSICAL TREATMENT; PRESERVATION OF FOODS OR FOODSTUFFS, IN GENERAL
    • 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
    • A23L33/17—Amino acids, peptides or proteins
    • A23L33/175—Amino acids
    • 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
    • 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
    • A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
    • A61K31/197—Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid, pantothenic acid
    • A61K31/198—Alpha-aminoacids, e.g. alanine, edetic acids [EDTA]
    • 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
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/5025—Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
    • 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
    • A61P3/00—Drugs for disorders of the metabolism

Abstract

The present invention is directed to a novel methods and compositions for the therapeutic intervention in hyperphenylalaninemia. More specifically, the specification describes methods and compositions for treating various types of phenylketonurias using compositions comprising BH4. Combination therapies of BH4 and other therapeutic regimens are contemplated.
PCT/US2004/038296 2003-11-17 2004-11-17 Treatment of phenylketonurias with bh4 WO2005049000A2 (en)

Priority Applications (16)

Application Number Priority Date Filing Date Title
DK04819152.2T DK1708690T3 (en) 2003-11-17 2004-11-17 TREATMENT OF PHENYLKETONURI WITH BH4
CA2545584A CA2545584C (en) 2003-11-17 2004-11-17 Methods and compositions for the treatment of metabolic disorders
JP2006539992A JP2007511536A (en) 2003-11-17 2004-11-17 Methods and compositions for the treatment of metabolic disorders
PL16178739T PL3138566T3 (en) 2003-11-17 2004-11-17 Treatment of phenylketonuria with bh4
BRPI0416566-7A BRPI0416566A (en) 2003-11-17 2004-11-17 phenylketonuria treatment with bh4
EP04819152.2A EP1708690B1 (en) 2003-11-17 2004-11-17 Treatment of phenylketonuria with bh4
EP21209606.9A EP3977999A1 (en) 2003-11-17 2004-11-17 Treatment of phenylketonuria with bh4
AU2004291149A AU2004291149C1 (en) 2003-11-17 2004-11-17 Methods and Compositions for the Treatment of Metabolic Disorders
ES04819152.2T ES2596827T3 (en) 2003-11-17 2004-11-17 Treatment of phenylketonuria with BH4
MXPA06005451A MXPA06005451A (en) 2003-11-17 2004-11-17 Treatment of phenylketonurias with bh4.
CN2004800338351A CN1905863B (en) 2003-11-17 2004-11-17 Methods and compositions for the treatment of metabolic disorders
EP16178739.5A EP3138566B1 (en) 2003-11-17 2004-11-17 Treatment of phenylketonuria with bh4
IL175529A IL175529A (en) 2003-11-17 2006-05-09 Tetrahydrobiopterin (bh4) or optionally a salt form thereof for use in the treatment of a subject with phenylketonuria(pku)
HK07102354.9A HK1094875A1 (en) 2003-11-17 2007-03-02 Treatment of phenylketonuria with bh4 bh4
CY20161101054T CY1118171T1 (en) 2003-11-17 2016-10-19 THERAPEUTIC TREATMENT OF BEN4
CY20221100068T CY1124931T1 (en) 2003-11-17 2022-01-27 TREATMENT OF PHENYL KETONOURIA WITH BH4

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US52076703P 2003-11-17 2003-11-17
US60/520,767 2003-11-17

Publications (2)

Publication Number Publication Date
WO2005049000A2 WO2005049000A2 (en) 2005-06-02
WO2005049000A3 true WO2005049000A3 (en) 2005-11-10

Family

ID=34619513

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2004/038296 WO2005049000A2 (en) 2003-11-17 2004-11-17 Treatment of phenylketonurias with bh4

Country Status (19)

Country Link
US (6) US7566714B2 (en)
EP (3) EP3977999A1 (en)
JP (2) JP2007511536A (en)
KR (1) KR20070005550A (en)
CN (1) CN1905863B (en)
AU (1) AU2004291149C1 (en)
BR (1) BRPI0416566A (en)
CA (1) CA2545584C (en)
CY (2) CY1118171T1 (en)
DK (2) DK1708690T3 (en)
ES (2) ES2905305T4 (en)
HK (1) HK1094875A1 (en)
HU (2) HUE029533T2 (en)
IL (1) IL175529A (en)
MX (1) MXPA06005451A (en)
PL (2) PL1708690T3 (en)
PT (2) PT1708690T (en)
SI (1) SI3138566T1 (en)
WO (1) WO2005049000A2 (en)

Cited By (1)

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US9216178B2 (en) 2011-11-02 2015-12-22 Biomarin Pharmaceutical Inc. Dry blend formulation of tetrahydrobiopterin

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PL1708690T3 (en) 2003-11-17 2017-01-31 Biomarin Pharmaceutical Inc. Treatment of phenylketonuria with bh4
WO2006055511A2 (en) * 2004-11-17 2006-05-26 Biomarin Pharmaceutical Inc. Stable tablet formulation of tetrahydrobiopterin
WO2007072911A1 (en) * 2005-12-22 2007-06-28 Asubio Pharma Co., Ltd. Preparation with improved bioabsorbability of sapropterin hydrochloride
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CA2675134A1 (en) * 2007-01-12 2008-07-24 Biomarin Pharmaceutical Inc. Pterin analogs
LT2545939T (en) 2007-04-11 2021-03-10 Biomarin Pharmaceutical Inc. Tetrahydrobiopterin for treating conditions associated with elevated phenylalanine levels
US20080275726A1 (en) * 2007-05-02 2008-11-06 Steven Yannicelli System and method for metabolic patient management and treatment
CA2711160A1 (en) 2008-01-03 2009-07-16 Biomarin Pharmaceutical Inc. Pterin analog for treating bh4 responsive condition
BRPI0907247A2 (en) * 2008-01-07 2015-07-14 Biomarin Pharm Inc Methods for preparing biopterin and for preparing tetrahydrobiopterin
UA102128C2 (en) * 2008-12-05 2013-06-10 Х. Луннбек А/С Nalmefene hydrochloride dihydrate
ES2669094T3 (en) * 2009-06-12 2018-05-24 Wisconsin Alumni Research Foundation Medicinal foods of glycomapropeptides for the nutritional treatment of phenylketonuria and other metabolic disorders
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EP2601946A1 (en) * 2011-12-06 2013-06-12 Pécsi Tudomànyegyetem Treatment and prevention of diseases related to oxidative stress
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CA2972638C (en) 2015-01-09 2023-11-28 Mcmaster University Allosteric activators for treatment of phenylketonuria, and method for identifying allosteric activators
US20180221371A1 (en) * 2015-08-07 2018-08-09 Children's National Medical Center Treatment for the fetus with congenital heart disease
JP2019527242A (en) 2016-07-29 2019-09-26 ディファーマ エッセ．ア． Pharmaceutical composition kit comprising sapropterin dihydrochloride
CN110312721A (en) * 2016-11-29 2019-10-08 显莎制药公司 The polymorph of sepiapterin
BR122021026889A8 (en) * 2016-11-29 2022-11-22 Censa Pharmaceuticals Inc FREE BASE CRYSTALLINE FORMS OF SEPIAPTERIN AND SALTS THEREOF, THEIR METHOD OF PREPARATION, COMPOSITIONS THAT COMPRISE THEM, USE OF THEM AND METHOD OF PREPARATION OF A LIQUID FORMULATION
JP2020532535A (en) 2017-09-01 2020-11-12 ピーティーシー セラピューティクス エムピー，インコーポレイテッド Pharmaceutical composition containing sepiapterin and its use
IT201700099690A1 (en) * 2017-09-06 2019-03-06 Abiogen Pharma Spa COMPOSITION FOR SOCCER INTEGRATION
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CN113727733B (en) * 2019-02-14 2023-01-24 索姆创新生物技术公司 Triamterene or nolatrexed for treating phenylketonuria
TR201914416A1 (en) * 2019-09-23 2021-04-21 Sanovel Ilac Sanayi Ve Ticaret Anonim Sirketi Effervescent formulations of sapropterin dihydrochloride
CN112362785B (en) * 2020-11-19 2022-12-27 南京工业大学 Application of a group of diagnostic markers in diagnosis of asthenospermia caused by varicocele
CN114569607B (en) * 2022-02-28 2024-02-13 军事科学院军事医学研究院环境医学与作业医学研究所 Application of 5-hydroxytryptophan in preparation of health care product or medicine for improving female physiological cycle disorder caused by altitude hypoxia

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