CN118055767A - 新颖的组合施用 - Google Patents

新颖的组合施用 Download PDF

Info

Publication number
CN118055767A
CN118055767A CN202280067699.6A CN202280067699A CN118055767A CN 118055767 A CN118055767 A CN 118055767A CN 202280067699 A CN202280067699 A CN 202280067699A CN 118055767 A CN118055767 A CN 118055767A
Authority
CN
China
Prior art keywords
seq
weight
sma
patient
sipu
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
CN202280067699.6A
Other languages
English (en)
Chinese (zh)
Inventor
尼科尔·黑尔巴赫
海德玛丽·克莱特兹尔
弗里德里希·梅茨格
雷娜塔·西奇利亚尼斯卡尔科
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.)
F Hoffmann La Roche AG
Original Assignee
F Hoffmann La Roche 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 F Hoffmann La Roche AG filed Critical F Hoffmann La Roche AG
Publication of CN118055767A publication Critical patent/CN118055767A/zh
Pending legal-status Critical Current

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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/12—Carboxylic acids; Salts or anhydrides thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A61K47/183—Amino acids, e.g. glycine, EDTA or aspartame
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0087—Galenical forms not covered by A61K9/02 - A61K9/7023
    • A61K9/0095—Drinks; Beverages; Syrups; Compositions for reconstitution thereof, e.g. powders or tablets to be dispersed in a glass of water; Veterinary drenches
    • 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
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/545—Medicinal preparations containing antigens or antibodies characterised by the dose, timing or administration schedule

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Mycology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Endocrinology (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
CN202280067699.6A 2021-10-06 2022-10-04 新颖的组合施用 Pending CN118055767A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP21201211.6 2021-10-06
EP21201211 2021-10-06
PCT/EP2022/077494 WO2023057404A1 (en) 2021-10-06 2022-10-04 Novel combined administration

Publications (1)

Publication Number Publication Date
CN118055767A true CN118055767A (zh) 2024-05-17

Family

ID=78085538

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202280067699.6A Pending CN118055767A (zh) 2021-10-06 2022-10-04 新颖的组合施用

Country Status (5)

Country Link
US (1) US20240358704A1 (https=)
EP (1) EP4426306A1 (https=)
JP (1) JP2024537864A (https=)
CN (1) CN118055767A (https=)
WO (1) WO2023057404A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118045206A (zh) * 2024-04-12 2024-05-17 四川至善唯新生物科技有限公司 一种治疗脊髓型肌肉萎缩的药物组合物及其用途

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4601651A1 (en) 2022-10-14 2025-08-20 Genentech, Inc. Methods for treating spinal muscular atrophy
US20250144031A1 (en) * 2023-10-27 2025-05-08 Hoffmann-La Roche Inc. Composition for treating spinal muscular atrophy

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6267958B1 (en) 1995-07-27 2001-07-31 Genentech, Inc. Protein formulation
US6171586B1 (en) 1997-06-13 2001-01-09 Genentech, Inc. Antibody formulation
JO3000B1 (ar) 2004-10-20 2016-09-05 Genentech Inc مركبات أجسام مضادة .
SI3143025T1 (sl) 2014-05-15 2020-01-31 F. Hoffmann-La Roche Ag Spojine za zdravljenje spinalne mišične atrofije
KR101860280B1 (ko) 2014-12-19 2018-05-21 추가이 세이야쿠 가부시키가이샤 항-마이오스타틴 항체, 변이체 Fc 영역을 함유하는 폴리펩타이드, 및 사용 방법
JP6659841B2 (ja) 2015-11-12 2020-03-04 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 脊髄性筋萎縮症を処置するための組成物
AR107078A1 (es) 2015-12-18 2018-03-21 Chugai Pharmaceutical Co Ltd Anticuerpo antimiostatina, polipéptidos que contienen regiones fc variantes así como métodos de uso
CN115814077A (zh) * 2016-01-08 2023-03-21 供石公司 抗-原肌生长抑制素/潜伏肌生长抑制素抗体及其使用方法
NZ789269A (en) * 2016-06-13 2026-01-30 Scholar Rock Inc Use of myostatin inhibitors and combination therapies
IL319888A (en) 2018-10-19 2025-05-01 Hoffmann La Roche Forms of pyrido[2,1-A]pyrimidin-4-one derivatives, their formulations and process for their preparation

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118045206A (zh) * 2024-04-12 2024-05-17 四川至善唯新生物科技有限公司 一种治疗脊髓型肌肉萎缩的药物组合物及其用途
CN118045206B (zh) * 2024-04-12 2024-07-05 四川至善唯新生物科技有限公司 一种治疗脊髓型肌肉萎缩的药物组合物及其用途

Also Published As

Publication number Publication date
JP2024537864A (ja) 2024-10-16
US20240358704A1 (en) 2024-10-31
WO2023057404A1 (en) 2023-04-13
EP4426306A1 (en) 2024-09-11

Similar Documents

Publication Publication Date Title
AU2020210134B2 (en) Use of myostatin inhibitors and combination therapies
JP5110877B2 (ja) 神経筋障害を処置するための、コルチコステロイドと組み合わせたミオスタチン（ｇｄｆ８）インヒビターの使用
TW202323296A (zh) 組合療法
CN118055767A (zh) 新颖的组合施用
EP4358995B1 (en) A myostatin pathway inhibitor in combination with a glp-1 pathway activator for use in treating metabolic disorders
JP2019504064A (ja) 抗プロ／潜在型ミオスタチン抗体およびその使用方法
EP4232151B1 (en) Use of anti-pro/latent myostatin antibody for treating spinal muscular atrophy
KR20150109386A (ko) 뒤시엔느 근위축증의 치료에서의 항-Flt-1 항체
JP2023541048A (ja) パーキンソン病の処置
JP2025514978A (ja) 脊髄性筋萎縮症を治療するためのミオスタチン阻害薬の使用
US20210101973A1 (en) Leap2 binding agents and compositions thereof
TW201204360A (en) Treatment of multiple sclerosis with MASITINIB
HK40103690A (zh) 新颖的组合施用
AU2023246859A1 (en) New treatment for facioscapulohumeral muscular dystrophy (fshd)
US20150297591A1 (en) Methods of treating pulmonary fibrosis
JP6429401B2 (ja) 変異を有するｉ型コラーゲンの小胞体内過剰蓄積正常化剤
RU2848183C1 (ru) Лечение болезни паркинсона
HK40112393A (zh) 用於面肩肱型肌营养不良症(fshd)的新治疗
JP2025524901A (ja) テストステロン及び成長ホルモンの組み合わせによる筋量、筋強度、又は筋機能を改善するための方法
EA051098B1 (ru) Применение антитела к промиостатину/латентному миостатину для лечения спинальной мышечной атрофии

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40103690

Country of ref document: HK

SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination