Use of migalastat for treating fabry disease in pregnant patients

A technology for migalastat and Fabry disease, applied to urinary system diseases, medical preparations containing active ingredients, pharmaceutical formulas, etc., can solve problems such as not recommending the use of migalastat

Pending Publication Date: 2020-11-20
AMICUS THERAPEUTICS INC
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  • Summary
  • Abstract
  • Description
  • Claims
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Problems solved by technology

However, developmental toxicity was observed when elevated doses were tested in pregnant rabbits
Although these doses are also maternally toxic, migalastat is not currently recommended for use during pregnancy

Method used

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  • Use of migalastat for treating fabry disease in pregnant patients
  • Use of migalastat for treating fabry disease in pregnant patients
  • Use of migalastat for treating fabry disease in pregnant patients

Examples

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example 1

[0129] Example 1: Patient treated with migalastat during pregnancy

[0130] In October 2005, a Caucasian female was diagnosed with Fabry disease based on renal biopsy and mutational analysis (GLA p.R112H). In 2009, the patient started treatment with alpha-galactosidase (ERT) for Fabry disease. After initiation of treatment, the patient has not seen progression of renal, cardiac, or neurologic symptoms.

[0131] In April 2012, the patient's proteinuria level was 187mg / 24h. In May 2012, the patient's proteinuria level was 83mg / 24h. In late May 2012, the 35-year-old patient began to participate in the phase 3 clinical trial of migalastat. The patient was also taking hormonal contraceptives.

[0132] This study describes a phase 3 study of migalastat therapy in Fabry patients who had undergone ERT. Eligible patients were aged 16-74 years and had genetically confirmed Fabry disease; had received ERT for ≥12 months; had a GLA mutation based on human embryonic kidney 293 (HEK)...

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Abstract

Provided are methods of treating a patient diagnosed with Fabry disease. Certain methods treat a pregnant patient with a therapeutically effective dose of migalastat or a salt thereof. Other methods treat a patient of childbearing potential with a therapeutically effective dose of migalastat or a salt thereof. Also described are the successful outcomes of pregnancies during which the pregnant patient is treated with migalastat.

Description

technical field [0001] The principles and embodiments of the present invention generally relate to the use of pharmacological partners for the treatment of Fabry disease, especially in pregnant women or patients of childbearing potential. [0002] Reference Sequence Listing [0003] The sequence listing text file submitted with this article is identified as "00790785.TXT" (22Kb, created on February 6, 2019), and is incorporated herein by reference. Background technique [0004] Many human diseases are caused by mutations that result in changes in the amino acid sequence of proteins that reduce protein stability and possibly prevent their proper folding. Proteins are normally folded in a specific region of the cell called the endoplasmic reticulum, or ER. Cells have quality control mechanisms that ensure that proteins are folded into their correct three-dimensional shape before they can move from the ER to their proper destination in the cell, a process commonly referred to...

Claims

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Application Information

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IPC IPC(8): A61K31/445A61P43/00
CPCA61K31/445A61P43/00A61P13/12A61K31/7008
Inventor 杰伊·巴斯
Owner AMICUS THERAPEUTICS INC
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