FI3801534T3 - Fenyyliketonurian hoito sepiapteriinilla - Google Patents

Fenyyliketonurian hoito sepiapteriinilla

Info

Publication number
FI3801534T3
FI3801534T3 FIEP19810058.8T FI19810058T FI3801534T3 FI 3801534 T3 FI3801534 T3 FI 3801534T3 FI 19810058 T FI19810058 T FI 19810058T FI 3801534 T3 FI3801534 T3 FI 3801534T3
Authority
FI
Finland
Prior art keywords
sepiapterin
pharmaceutically acceptable
acceptable salt
use according
administration
Prior art date
Application number
FIEP19810058.8T
Other languages
English (en)
Finnish (fi)
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 FI3801534T3 publication Critical patent/FI3801534T3/fi

Links

Classifications

    • 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
    • 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
    • A61K31/4985—Pyrazines or piperazines 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
    • 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
    • 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
    • A61K9/0065—Forms 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)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Epidemiology (AREA)
  • Physiology (AREA)
  • Nutrition Science (AREA)
  • Obesity (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
FIEP19810058.8T 2018-05-30 2019-05-30 Fenyyliketonurian hoito sepiapteriinilla FI3801534T3 (fi)

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
FI3801534T3 true FI3801534T3 (fi) 2026-04-14

Family

ID=68699016

Family Applications (1)

Application Number Title Priority Date Filing Date
FIEP19810058.8T FI3801534T3 (fi) 2018-05-30 2019-05-30 Fenyyliketonurian hoito sepiapteriinilla

Country Status (10)

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

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA46973A (fr) 2016-11-29 2019-10-09 Censa Pharmaceuticals Inc Polymorphes de sépiaptérine et leurs sels
MA46972A (fr) 2016-11-29 2019-10-09 Censa Pharmaceuticals Inc Forme polymorphe de sépiaptérine
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
FI3801536T3 (fi) * 2018-05-30 2024-08-29 Ptc Therapeutics Mp Inc Sepiapteriinin antaminen ilman ruokaa käytettäväksi menetelmässä sepiapteriinin plasma-altistuksen lisäämiseksi
EP3801534B1 (en) 2018-05-30 2026-02-18 PTC Therapeutics MP, Inc. Treatment of phenylketonuria with sepiapterin
EP4093405A1 (en) * 2020-01-24 2022-11-30 PTC Therapeutics MP, Inc. Methods for treating parkinson's disease with sepiapterin

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
JP2002515965A (ja) 1995-08-18 2002-05-28 ダブリュー． ランドリ，ドナルド 抗体触媒反応の調節による有機化合物の検出
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
WO2005048926A2 (en) 2003-11-13 2005-06-02 The General Hospital Corporation Methods for treating pain
PL1708690T3 (pl) 2003-11-17 2017-01-31 Biomarin Pharmaceutical Inc. Leczenie fenyloketonurii za pomocą BH4
JP5184783B2 (ja) 2003-11-17 2013-04-17 バイオマリン ファーマシューティカル インコーポレイテッド テトラヒドロビオプテリン、及びテトラヒドロビオプテリン類似体の製造方法
EP1687308B1 (en) 2003-11-17 2019-06-12 Merck & Cie Process for preparating (6R)-L-erythrotetrahydrobiopterin hydrochloride crystalline form B from other crystalline forms
KR20070084270A (ko) 2004-11-17 2007-08-24 바이오마린 파머수티컬 인크. 안정한 정제 제형물
JP2008523090A (ja) 2004-12-08 2008-07-03 バイオマリン ファーマシューティカル インコーポレイテッド 新生児肺高血圧症の治療のための方法および組成物
BRPI0609421B1 (pt) 2005-04-28 2021-06-01 Shiratori Pharmaceutical Co., Ltd. Processos para produção de um composto 1,1-bis-alquil sulfonila, para produção de um composto aldeído, e para produção de um derivado de hidrazona
US20080075666A1 (en) 2006-08-25 2008-03-27 Dudley Samuel C Jr Methods and compositions for treating diastolic dysfunction
LT2545939T (lt) 2007-04-11 2021-03-10 Biomarin Pharmaceutical Inc. Tetrahidrobiopterinas, skirtas gydyti ligas, susijusias supadidėjusiu fenilalanino kiekiu
WO2010017570A2 (de) 2008-08-12 2010-02-18 Orpha Swiss Gmbh Pharmazeutische darreichungsform enthaltend 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
CN103458900A (zh) * 2011-03-01 2013-12-18 鲁必康研究私人有限公司 四氢生物蝶呤的稳定组合物
US9216178B2 (en) * 2011-11-02 2015-12-22 Biomarin Pharmaceutical Inc. Dry blend formulation of tetrahydrobiopterin
CN104736539B (zh) 2012-05-07 2017-09-01 白鸟制药株式会社 墨蝶呤和四氢乳酰蝶呤的制造法
EP3213062B1 (en) 2014-10-31 2020-02-12 Children's Medical Center Corporation Methods and assays relating to sepiapterin reductase inhibition
MA46972A (fr) 2016-11-29 2019-10-09 Censa Pharmaceuticals Inc Forme polymorphe de sépiaptérine
MA46973A (fr) 2016-11-29 2019-10-09 Censa Pharmaceuticals Inc Polymorphes de sépiaptérine et leurs sels
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
EP3801534B1 (en) 2018-05-30 2026-02-18 PTC Therapeutics MP, Inc. Treatment of phenylketonuria with sepiapterin
FI3801536T3 (fi) 2018-05-30 2024-08-29 Ptc Therapeutics Mp Inc Sepiapteriinin antaminen ilman ruokaa käytettäväksi menetelmässä sepiapteriinin plasma-altistuksen lisäämiseksi
AU2019277372B2 (en) * 2018-05-30 2024-12-19 Ptc Therapeutics Mp, Inc. Pharmaceutically acceptable salts of sepiapterin
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
WO2019232126A1 (en) 2019-12-05
JP7547215B2 (ja) 2024-09-09
US12257252B2 (en) 2025-03-25
EP3801534B1 (en) 2026-02-18
EP3801534A4 (en) 2022-03-16
EP3801534A1 (en) 2021-04-14
JP2024178188A (ja) 2024-12-24
US20250195529A1 (en) 2025-06-19
DK3801534T3 (da) 2026-03-30
US20210161901A1 (en) 2021-06-03
MX2020012980A (es) 2021-04-29
CA3102105A1 (en) 2019-12-05
CN112654356A (zh) 2021-04-13
BR112020024329A2 (pt) 2021-02-23
JP2021525746A (ja) 2021-09-27

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