DK3801534T3 - Behandling af fenylketonuri med sepiapterin - Google Patents

Behandling af fenylketonuri med sepiapterin

Info

Publication number
DK3801534T3
DK3801534T3 DK19810058.8T DK19810058T DK3801534T3 DK 3801534 T3 DK3801534 T3 DK 3801534T3 DK 19810058 T DK19810058 T DK 19810058T DK 3801534 T3 DK3801534 T3 DK 3801534T3
Authority
DK
Denmark
Prior art keywords
sepiapterin
phenylketonuria
treatment
Prior art date
Application number
DK19810058.8T
Other languages
Danish (da)
English (en)
Inventor
Neil Smith
Jonathan Reis
Original Assignee
Ptc Therapeutics Mp 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
Application filed by Ptc Therapeutics Mp Inc filed Critical Ptc Therapeutics Mp Inc
Application granted granted Critical
Publication of DK3801534T3 publication Critical patent/DK3801534T3/da

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/4985Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration
    • A61K9/0065Forms with gastric retention, e.g. floating on gastric juice, adhering to gastric mucosa, expanding to prevent passage through the pylorus

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
DK19810058.8T 2018-05-30 2019-05-30 Behandling af fenylketonuri med sepiapterin DK3801534T3 (da)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862677926P 2018-05-30 2018-05-30
US201862771397P 2018-11-26 2018-11-26
US201962822365P 2019-03-22 2019-03-22
GC201937662 2019-05-28
PCT/US2019/034515 WO2019232126A1 (en) 2018-05-30 2019-05-30 Compositions and methods for increasing tetrahydrobiopterin plasma exposure

Publications (1)

Publication Number Publication Date
DK3801534T3 true DK3801534T3 (da) 2026-03-30

Family

ID=68699016

Family Applications (1)

Application Number Title Priority Date Filing Date
DK19810058.8T DK3801534T3 (da) 2018-05-30 2019-05-30 Behandling af fenylketonuri med sepiapterin

Country Status (10)

Country Link
US (2) US12257252B2 (https=)
EP (1) EP3801534B1 (https=)
JP (2) JP7547215B2 (https=)
CN (1) CN112654356A (https=)
BR (1) BR112020024329A2 (https=)
CA (1) CA3102105A1 (https=)
DK (1) DK3801534T3 (https=)
FI (1) FI3801534T3 (https=)
MX (1) MX2020012980A (https=)
WO (1) WO2019232126A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11072614B2 (en) 2016-11-29 2021-07-27 Ptc Therapeutics Mp, Inc. Polymorphs of sepiapterin and salts thereof
US11130760B2 (en) 2016-11-29 2021-09-28 Ptc Therapeutics Mp, Inc. Polymorphic form of sepiapterin
JP7811085B2 (ja) 2017-09-01 2026-02-04 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 セピアプテリンを含む医薬組成物及びその使用
EP3801536B1 (en) * 2018-05-30 2024-07-17 PTC Therapeutics MP, Inc. Administration of sepiapterin without food for use in a method for increasing sepiapterin plasma exposure
CN112654356A (zh) 2018-05-30 2021-04-13 Ptc医疗Mp公司 增加四氢生物蝶呤血浆暴露的组合物和方法
CA3165636A1 (en) * 2020-01-24 2021-07-29 Neil Smith Methods for treating parkinson's disease with sepiapterin
US12298026B1 (en) 2021-04-12 2025-05-13 W. L. Gore & Associates, Inc. Latent energy transfer laminate for plate pack core

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US5910304A (en) 1982-12-13 1999-06-08 Texas A&M University System Low-dose oral administration of interferons
CA1320905C (en) 1986-11-06 1993-08-03 Joseph M. Cummins Treatment of immuno-resistant disease
CA2228970A1 (en) 1995-08-18 1997-02-27 Donald W. Landry Detection of organic compounds through regulation of antibody-catalyzed reactions
US6036949A (en) 1998-03-05 2000-03-14 Amarillo Biosciences, Inc. Treatment of fibromyalgia with low doses of interferon
US8188043B2 (en) 1999-07-28 2012-05-29 The Board Of Trustees Of The Leland Stanford Jr. University Nicotine in therapeutic angiogenesis and vasculogenesis
FR2859996B1 (fr) 2003-09-19 2006-02-03 Aventis Pharma Sa Solvat acetonique du dimethoxy docetaxel et son procede de preparation
EP1696877A4 (en) 2003-11-13 2010-06-09 Gen Hospital Corp PROCESS FOR PAIN TREATMENT
US7732599B2 (en) 2003-11-17 2010-06-08 Biomarin Pharmaceutical Inc. Process for preparing tetrahydrobiopterin and analogs of tetrahydrobiopterin
CA2678125C (en) 2003-11-17 2014-10-14 Merck Eprova Ag Crystalline forms of (6r)-l-erythro-tetrahydrobiopterin dihydrochloride
KR20070005550A (ko) 2003-11-17 2007-01-10 바이오마린 파머수티컬 인크. 대사 장애 치료를 위한 조성물 및 방법
WO2006055511A2 (en) 2004-11-17 2006-05-26 Biomarin Pharmaceutical Inc. Stable tablet formulation of tetrahydrobiopterin
CA2588994A1 (en) 2004-12-08 2006-06-15 Biomarin Pharmaceutical Inc. Methods and compositions for the treatment of pulmonary hypertension of the newborn
US7582799B2 (en) 2005-04-28 2009-09-01 Shiratori Pharmaceutical Co., Ltd. Method for producing hydrazone derivatives
US20080075666A1 (en) 2006-08-25 2008-03-27 Dudley Samuel C Jr Methods and compositions for treating diastolic dysfunction
WO2008128049A2 (en) 2007-04-11 2008-10-23 Biomarin Pharmaceutical Inc. Methods of administering tetrahydrobiopterin, associated compositions, and methods of measuring
US20110144117A1 (en) 2008-08-12 2011-06-16 Orpha Swiss Gmbh Pharmaceutical Dosage Form Containing Tetrahydrobiopterin
EP2562176B1 (en) 2010-04-22 2018-08-15 Nihon University Medicinal agent and beverage/food for preventing cerebral dysfunction and improving same
US9884909B2 (en) 2010-12-01 2018-02-06 Alderbio Holdings Llc Anti-NGF compositions and use thereof
CA2828685C (en) 2011-03-01 2018-05-29 Rubicon Research Private Limited Stable compositions of tetrahydrobiopterin
US9216178B2 (en) * 2011-11-02 2015-12-22 Biomarin Pharmaceutical Inc. Dry blend formulation of tetrahydrobiopterin
IN2014DN09053A (https=) 2012-05-07 2015-05-22 Shiratori Pharm
US10365267B2 (en) 2014-10-31 2019-07-30 Children's Medical Center Corporation Methods and assays relating to sepiapterin reductase inhibition
US11130760B2 (en) 2016-11-29 2021-09-28 Ptc Therapeutics Mp, Inc. Polymorphic form of sepiapterin
US11072614B2 (en) * 2016-11-29 2021-07-27 Ptc Therapeutics Mp, Inc. Polymorphs of sepiapterin and salts thereof
JP7811085B2 (ja) 2017-09-01 2026-02-04 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 セピアプテリンを含む医薬組成物及びその使用
CN112654356A (zh) 2018-05-30 2021-04-13 Ptc医疗Mp公司 增加四氢生物蝶呤血浆暴露的组合物和方法
AU2019277372B2 (en) * 2018-05-30 2024-12-19 Ptc Therapeutics Mp, Inc. Pharmaceutically acceptable salts of sepiapterin
EP3801536B1 (en) 2018-05-30 2024-07-17 PTC Therapeutics MP, Inc. Administration of sepiapterin without food for use in a method for increasing sepiapterin plasma exposure
IL290321B1 (en) 2019-08-05 2026-04-01 Ptc Therapeutics Inc Use of spiapterin and its metabolites for radiation exposure treatment
IL291565B1 (en) 2019-09-25 2026-04-01 Ptc Therapeutics Inc Method for treating phenylalanine irregularity

Also Published As

Publication number Publication date
JP2024178188A (ja) 2024-12-24
FI3801534T3 (fi) 2026-04-14
US20210161901A1 (en) 2021-06-03
US12257252B2 (en) 2025-03-25
JP2021525746A (ja) 2021-09-27
EP3801534B1 (en) 2026-02-18
BR112020024329A2 (pt) 2021-02-23
CN112654356A (zh) 2021-04-13
JP7547215B2 (ja) 2024-09-09
US20250195529A1 (en) 2025-06-19
MX2020012980A (es) 2021-04-29
CA3102105A1 (en) 2019-12-05
EP3801534A4 (en) 2022-03-16
EP3801534A1 (en) 2021-04-14
WO2019232126A1 (en) 2019-12-05

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