WO2019152343A8 - Methods of desensitizing against infusion associated reactions in lysosomal acid lipase deficiency (lal-d) patients treated with exogenous lysosomal acid lipase (lal) - Google Patents

Methods of desensitizing against infusion associated reactions in lysosomal acid lipase deficiency (lal-d) patients treated with exogenous lysosomal acid lipase (lal) Download PDF

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Publication number
WO2019152343A8
WO2019152343A8 PCT/US2019/015524 US2019015524W WO2019152343A8 WO 2019152343 A8 WO2019152343 A8 WO 2019152343A8 US 2019015524 W US2019015524 W US 2019015524W WO 2019152343 A8 WO2019152343 A8 WO 2019152343A8
Authority
WO
WIPO (PCT)
Prior art keywords
lal
acid lipase
lysosomal acid
methods
exogenous
Prior art date
Application number
PCT/US2019/015524
Other languages
French (fr)
Other versions
WO2019152343A1 (en
Inventor
Mark Friedman
Suresh VIJAY
Julian RAIMAN
Srividya SREEKANTAM
Original Assignee
Alexion Pharmaceuticals, 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 Alexion Pharmaceuticals, Inc. filed Critical Alexion Pharmaceuticals, Inc.
Publication of WO2019152343A1 publication Critical patent/WO2019152343A1/en
Publication of WO2019152343A8 publication Critical patent/WO2019152343A8/en

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01013—Sterol esterase (3.1.1.13)

Abstract

The disclosure features methods for safely and effectively administering exogenous LAL (e.g., sebelipase alfa) to pediatric LAL-D patients, as well as methods to prevent and ameliorate infusion reactions in LAL-D pediatric patients receiving exogenous LAL. In particular, disclosed herein are specific dosages and regimens for administering exogenous LAL to a pediatric patient having LAL-D, such dosages and regimens being designed to increase a patient's tolerance of the therapeutic, decrease the likelihood of (e.g., desensitize against) an infusion-related reaction, and maximize the amount of dose that may be administered to the patient safely, so as to safely achieve maximum therapeutic benefits.
PCT/US2019/015524 2018-02-01 2019-01-29 Methods of desensitizing against infusion associated reactions in lysosomal acid lipase deficiency (lal-d) patients treated with exogenous lysosomal acid lipase (lal WO2019152343A1 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862624890P 2018-02-01 2018-02-01
US62/624,890 2018-02-01
US201862768524P 2018-11-16 2018-11-16
US62/768,524 2018-11-16

Publications (2)

Publication Number Publication Date
WO2019152343A1 WO2019152343A1 (en) 2019-08-08
WO2019152343A8 true WO2019152343A8 (en) 2019-09-12

Family

ID=65409581

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2019/015524 WO2019152343A1 (en) 2018-02-01 2019-01-29 Methods of desensitizing against infusion associated reactions in lysosomal acid lipase deficiency (lal-d) patients treated with exogenous lysosomal acid lipase (lal

Country Status (1)

Country Link
WO (1) WO2019152343A1 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023505718A (en) * 2019-12-11 2023-02-10 サドレア ラボラトリーズ プロプライエタリー リミテッド Drug delivery system and method

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2006302640B2 (en) 2005-10-05 2010-08-19 Synageva Biopharma Corp. Rapid production of high titer virus
LT3205351T (en) 2010-04-23 2023-07-25 Alexion Pharmaceuticals, Inc. Lysosomal storage disease enzyme

Also Published As

Publication number Publication date
WO2019152343A1 (en) 2019-08-08

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