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Alpha-galactosidase A

2

Histamine H1 receptor

316

Dopamine D2 receptor

311

DNA

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Serotonin 2a (5-HT2a) receptor

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2018

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Fabry's disease

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Primary

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Amicus Therapeutics

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Chemical

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CAS

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ChEMBL

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Various alimentary tract and metabolism products

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MIGALASTAT

C4XNY919FW

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Status:

US Approved Rx (2018)

First approved in 2018

Class (Stereo):

CHEMICAL (ABSOLUTE)

Targets:

Alpha-galactosidase A

Conditions:

Fabry's disease

Migalastat (Galafold)-a small molecule drug developed by Amicus Therapeutics that restores the activity of specific mutant forms of α-galactosidase-has been approved for the treatment of Fabry disease in the EU in patients with amenable mutations. Mi...

galastat attaches to certain unstable forms of alpha-galactosidase A, stabilising the enzyme. This allows the enzyme to be transported into areas of the cell where it can break down GL-3. Under the trade name Galafold (formerly known as Amigal), Migalastat is used to treat patients aged 16 years or over with Fabry disease. Because the number of patients with Fabry disease is low, the disease is considered ‘rare’, and the US Food and Drug Administration (FDA) assigned Galafold orphan drug status in 2004, and the European Committee for Medicinal Products for Human Use (CHMP) followed in 2006.

MIGALASTAT HYDROCHLORIDE

CLY7M0XD20

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Status:

US Approved Rx (2018)

First approved in 2018

Class (Stereo):

CHEMICAL (ABSOLUTE)

Targets:

Alpha-galactosidase A

Conditions:

Fabry's disease

Migalastat (Galafold)-a small molecule drug developed by Amicus Therapeutics that restores the activity of specific mutant forms of α-galactosidase-has been approved for the treatment of Fabry disease in the EU in patients with amenable mutations. Mi...

galastat attaches to certain unstable forms of alpha-galactosidase A, stabilising the enzyme. This allows the enzyme to be transported into areas of the cell where it can break down GL-3. Under the trade name Galafold (formerly known as Amigal), Migalastat is used to treat patients aged 16 years or over with Fabry disease. Because the number of patients with Fabry disease is low, the disease is considered ‘rare’, and the US Food and Drug Administration (FDA) assigned Galafold orphan drug status in 2004, and the European Committee for Medicinal Products for Human Use (CHMP) followed in 2006.

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