CA3102105A1 - Compositions and methods for increasing tetrahydrobiopterin plasma exposure - Google Patents

Compositions and methods for increasing tetrahydrobiopterin plasma exposure Download PDF

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Publication number
CA3102105A1
CA3102105A1 CA3102105A CA3102105A CA3102105A1 CA 3102105 A1 CA3102105 A1 CA 3102105A1 CA 3102105 A CA3102105 A CA 3102105A CA 3102105 A CA3102105 A CA 3102105A CA 3102105 A1 CA3102105 A1 CA 3102105A1
Authority
CA
Canada
Prior art keywords
sepiapterin
subject
food
administration
effective amount
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.)
Pending
Application number
CA3102105A
Other languages
English (en)
French (fr)
Inventor
Neil Smith
Jonathan Reis
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.)
PTC Therapeutics MP Inc
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
Publication of CA3102105A1 publication Critical patent/CA3102105A1/en
Pending legal-status Critical Current

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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
    • 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
    • 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)
  • Public Health (AREA)
  • Veterinary Medicine (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)
  • 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)
CA3102105A 2018-05-30 2019-05-30 Compositions and methods for increasing tetrahydrobiopterin plasma exposure Pending CA3102105A1 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862677926P 2018-05-30 2018-05-30
US62/677,926 2018-05-30
US201862771397P 2018-11-26 2018-11-26
US62/771,397 2018-11-26
US201962822365P 2019-03-22 2019-03-22
US62/822,365 2019-03-22
GC2019/37662 2019-05-28
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
CA3102105A1 true CA3102105A1 (en) 2019-12-05

Family

ID=68699016

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3102105A Pending CA3102105A1 (en) 2018-05-30 2019-05-30 Compositions and methods for increasing tetrahydrobiopterin plasma exposure

Country Status (22)

Country Link
US (2) US12257252B2 (cg-RX-API-DMAC10.html)
EP (2) EP3801534B1 (cg-RX-API-DMAC10.html)
JP (2) JP7547215B2 (cg-RX-API-DMAC10.html)
CN (1) CN112654356A (cg-RX-API-DMAC10.html)
BR (1) BR112020024329A2 (cg-RX-API-DMAC10.html)
CA (1) CA3102105A1 (cg-RX-API-DMAC10.html)
DK (1) DK3801534T3 (cg-RX-API-DMAC10.html)
ES (1) ES3068414T3 (cg-RX-API-DMAC10.html)
FI (1) FI3801534T3 (cg-RX-API-DMAC10.html)
FR (1) FR26C1028I1 (cg-RX-API-DMAC10.html)
HR (1) HRP20260360T1 (cg-RX-API-DMAC10.html)
LT (2) LT3801534T (cg-RX-API-DMAC10.html)
MD (1) MD3801534T2 (cg-RX-API-DMAC10.html)
MX (1) MX2020012980A (cg-RX-API-DMAC10.html)
NL (1) NL301382I2 (cg-RX-API-DMAC10.html)
NO (1) NO2026028I1 (cg-RX-API-DMAC10.html)
PL (1) PL3801534T3 (cg-RX-API-DMAC10.html)
PT (1) PT3801534T (cg-RX-API-DMAC10.html)
RS (1) RS68045B1 (cg-RX-API-DMAC10.html)
SI (1) SI3801534T1 (cg-RX-API-DMAC10.html)
SM (1) SMT202600220T1 (cg-RX-API-DMAC10.html)
WO (1) WO2019232126A1 (cg-RX-API-DMAC10.html)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
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CN110312721A (zh) 2016-11-29 2019-10-08 显莎制药公司 墨蝶呤的多晶型物
WO2018102315A1 (en) 2016-11-29 2018-06-07 Censa Pharmaceuticals Inc. Polymoprhs of sepiapterin and salts thereof
MX2020002271A (es) 2017-09-01 2020-10-05 Ptc Therapeutics Mp Inc Composiciones farmaceuticas que comprenden sepiapterina y sus usos de las mismas.
CA3102105A1 (en) 2018-05-30 2019-12-05 Ptc Therapeutics Mp, Inc. Compositions and methods for increasing tetrahydrobiopterin plasma exposure
JP7778477B2 (ja) 2018-05-30 2025-12-02 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 セピアプテリンの薬学的に許容される塩
JP7502197B2 (ja) * 2018-05-30 2024-06-18 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 セピアプテリン血漿曝露を増加させるための方法
LT4009978T (lt) 2019-08-05 2024-11-25 Ptc Therapeutics Mp, Inc. Sepiapterino ir jo metabolitų panaudojimas spinduliuotės poveikio gydymui
CA3165636A1 (en) * 2020-01-24 2021-07-29 Neil Smith Methods for treating parkinson's disease with sepiapterin
US12691119B2 (en) 2021-02-09 2026-07-28 Ptc Therapeutics Mp, Inc. Methods for treating covid-19 with sepiapterin
US12298026B1 (en) 2021-04-12 2025-05-13 W. L. Gore & Associates, Inc. Latent energy transfer laminate for plate pack core

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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
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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
CA2545968C (en) 2003-11-17 2010-03-09 Merck Eprova Ag Crystalline forms of (6r)-l-erythro-tetrahydrobiopterin dihydrochloride
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
PT3138566T (pt) 2003-11-17 2022-02-02 Biomarin Pharm Inc Tratamento de fenilcetonúria com bh4
CN101132776A (zh) 2004-11-17 2008-02-27 生物马林药物股份有限公司 稳定的四氢生物喋呤片剂制剂
US20100130500A1 (en) 2004-12-08 2010-05-27 Biomarin Pharmaceutical Inc. Methods and compositions for the treatment of pulmonary hypertension of the newborn
WO2006118322A1 (en) 2005-04-28 2006-11-09 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
KR20160065998A (ko) * 2007-04-11 2016-06-09 바이오마린 파머수티컬 인크. 테트라하이드로바이오프테린 투여 방법, 관련 조성물 및 측정 방법
WO2010017570A2 (de) 2008-08-12 2010-02-18 Orpha Swiss Gmbh Pharmazeutische darreichungsform enthaltend tetrahydrobiopterin
JP5066756B2 (ja) * 2010-04-22 2012-11-07 学校法人日本大学 脳機能障害予防・改善用の薬剤及び飲食物
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US9216178B2 (en) * 2011-11-02 2015-12-22 Biomarin Pharmaceutical Inc. Dry blend formulation of tetrahydrobiopterin
JP6084967B2 (ja) 2012-05-07 2017-02-22 白鳥製薬株式会社 セピアプテリン及びテトラヒドロラクトイルプテリンの製造法
EP3213062B1 (en) 2014-10-31 2020-02-12 Children's Medical Center Corporation Methods and assays relating to sepiapterin reductase inhibition
WO2018102315A1 (en) * 2016-11-29 2018-06-07 Censa Pharmaceuticals Inc. Polymoprhs of sepiapterin and salts thereof
CN110312721A (zh) 2016-11-29 2019-10-08 显莎制药公司 墨蝶呤的多晶型物
MX2020002271A (es) * 2017-09-01 2020-10-05 Ptc Therapeutics Mp Inc Composiciones farmaceuticas que comprenden sepiapterina y sus usos de las mismas.
JP7778477B2 (ja) * 2018-05-30 2025-12-02 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 セピアプテリンの薬学的に許容される塩
CA3102105A1 (en) 2018-05-30 2019-12-05 Ptc Therapeutics Mp, Inc. Compositions and methods for increasing tetrahydrobiopterin plasma exposure
JP7502197B2 (ja) 2018-05-30 2024-06-18 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 セピアプテリン血漿曝露を増加させるための方法
LT4009978T (lt) 2019-08-05 2024-11-25 Ptc Therapeutics Mp, Inc. Sepiapterino ir jo metabolitų panaudojimas spinduliuotės poveikio gydymui
IL291565B1 (en) 2019-09-25 2026-04-01 Ptc Therapeutics Inc Method for treating phenylalanine irregularity

Also Published As

Publication number Publication date
EP4748370A2 (en) 2026-05-27
PL3801534T3 (pl) 2026-07-06
FR26C1028I1 (fr) 2026-07-17
JP7547215B2 (ja) 2024-09-09
JP2021525746A (ja) 2021-09-27
EP4748370A3 (en) 2026-07-29
ES3068414T3 (en) 2026-05-29
EP3801534A1 (en) 2021-04-14
SMT202600220T1 (it) 2026-07-16
JP7855033B2 (ja) 2026-05-07
US20210161901A1 (en) 2021-06-03
SI3801534T1 (sl) 2026-06-30
LTPA2026521I1 (cg-RX-API-DMAC10.html) 2026-06-10
US20250195529A1 (en) 2025-06-19
JP2024178188A (ja) 2024-12-24
NO2026028I1 (no) 2026-05-29
MX2020012980A (es) 2021-04-29
CN112654356A (zh) 2021-04-13
FI3801534T3 (fi) 2026-04-14
LT3801534T (lt) 2026-05-25
NL301382I2 (nl) 2026-07-16
EP3801534B1 (en) 2026-02-18
MD3801534T2 (en) 2026-07-31
US12257252B2 (en) 2025-03-25
EP3801534A4 (en) 2022-03-16
WO2019232126A1 (en) 2019-12-05
RS68045B1 (sr) 2026-05-29
HRP20260360T1 (hr) 2026-06-05
DK3801534T3 (da) 2026-03-30
BR112020024329A2 (pt) 2021-02-23
PT3801534T (pt) 2026-04-28

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