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US Approved Rx

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Primary Target

Pyruvate kinase isozymes M1/M2

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Approval Year

2022

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Unknown

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Originator

Agios Pharmaceuticals

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Chemical

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PUBCHEM

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FDA ORPHAN DRUG

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Showing 1 - 3 of 3 results

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MITAPIVAT

2WTV10SIKH

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Status:

US Approved Rx (2022)

First approved in 2022

Class (Stereo):

CHEMICAL (ACHIRAL)

Targets:

Pyruvate kinase PKLR

Pyruvate kinase isozymes M1/M2

Conditions:

Hemolytic anemia due to pyruvate kinase deficiency

Mitapivat (AG-348; PKM2 activator 1020) is a novel, first-in-class oral small molecule allosteric activator of the pyruvate kinase enzyme. Mitapivat has been shown to significantly upregulate both wild-type and numerous mutant forms of erythrocyte py...

ruvate kinase (PKR), increasing adenosine triphosphate (ATP) production and reducing levels of 2,3-diphosphoglycerate. Given this mechanism, mitapivat has been evaluated in clinical trials in a wide range of hereditary hemolytic anemias, including pyruvate kinase deficiency (PKD), sickle cell disease, and the thalassemias. Mitapivat was approved for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the United States in February 2022, and in the European Union in November 2022.

MITAPIVAT SULFATE ANHYDROUS

KM3KSE3QH9

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Status:

US Approved Rx (2022)

First approved in 2022

Class (Stereo):

CHEMICAL (ACHIRAL)

Targets:

Pyruvate kinase PKLR

Pyruvate kinase isozymes M1/M2

Conditions:

Hemolytic anemia due to pyruvate kinase deficiency

Mitapivat (AG-348; PKM2 activator 1020) is a novel, first-in-class oral small molecule allosteric activator of the pyruvate kinase enzyme. Mitapivat has been shown to significantly upregulate both wild-type and numerous mutant forms of erythrocyte py...

ruvate kinase (PKR), increasing adenosine triphosphate (ATP) production and reducing levels of 2,3-diphosphoglycerate. Given this mechanism, mitapivat has been evaluated in clinical trials in a wide range of hereditary hemolytic anemias, including pyruvate kinase deficiency (PKD), sickle cell disease, and the thalassemias. Mitapivat was approved for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the United States in February 2022, and in the European Union in November 2022.

MITAPIVAT SULFATE

N4JTA67V3O

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Search for Structure

Status:

US Approved Rx (2022)

First approved in 2022

Class (Stereo):

CHEMICAL (ACHIRAL)

Targets:

Pyruvate kinase PKLR

Pyruvate kinase isozymes M1/M2

Conditions:

Hemolytic anemia due to pyruvate kinase deficiency

Mitapivat (AG-348; PKM2 activator 1020) is a novel, first-in-class oral small molecule allosteric activator of the pyruvate kinase enzyme. Mitapivat has been shown to significantly upregulate both wild-type and numerous mutant forms of erythrocyte py...

ruvate kinase (PKR), increasing adenosine triphosphate (ATP) production and reducing levels of 2,3-diphosphoglycerate. Given this mechanism, mitapivat has been evaluated in clinical trials in a wide range of hereditary hemolytic anemias, including pyruvate kinase deficiency (PKD), sickle cell disease, and the thalassemias. Mitapivat was approved for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the United States in February 2022, and in the European Union in November 2022.

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