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Primary Target

Molybdopterin synthase catalytic subunit

3

Histamine H1 receptor

316

Dopamine D2 receptor

311

DNA

282

Serotonin 2a (5-HT2a) receptor

237

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US Approved Rx

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2021

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molybdenum cofactor deficiency Type A

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Showing 1 - 3 of 3 results

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FOSDENOPTERIN

4X7K2681Y7

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Status:

US Approved Rx (2021)

First approved in 2021

Class (Stereo):

CHEMICAL (ABSOLUTE)

Targets:

Molybdopterin synthase catalytic subunit

Conditions:

molybdenum cofactor deficiency Type A

Fosdenopterin (NulibryTM) is a synthetic cyclic pyranopterin monophosphate that is being developed by Origin Biosciences (a subsidiary of BridgeBio Pharma) for the treatment of molybdenum cofactor deficiency (MoCD) type A. Patients with MoCD Type A h...

ave mutations in the MOCS1 gene leading to deficient MOCS1A/B dependent synthesis of the intermediate substrate, cPMP. Substrate replacement therapy with NULIBRY provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase (SOX), an enzyme that reduces levels of neurotoxic sulfites. Fosdenopterin was approved by the US FDA in February 2021 for use in reducing the risk of mortality in paediatric and adult patients with MoCD type A.

FOSDENOPTERIN HYDROBROMIDE

X41B5W735T

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Status:

US Approved Rx (2021)

First approved in 2021

Class (Stereo):

CHEMICAL (ABSOLUTE)

Targets:

Molybdopterin synthase catalytic subunit

Conditions:

molybdenum cofactor deficiency Type A

Fosdenopterin (NulibryTM) is a synthetic cyclic pyranopterin monophosphate that is being developed by Origin Biosciences (a subsidiary of BridgeBio Pharma) for the treatment of molybdenum cofactor deficiency (MoCD) type A. Patients with MoCD Type A h...

ave mutations in the MOCS1 gene leading to deficient MOCS1A/B dependent synthesis of the intermediate substrate, cPMP. Substrate replacement therapy with NULIBRY provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase (SOX), an enzyme that reduces levels of neurotoxic sulfites. Fosdenopterin was approved by the US FDA in February 2021 for use in reducing the risk of mortality in paediatric and adult patients with MoCD type A.

FOSDENOPTERIN HYDROBROMIDE ANHYDROUS

8Y2YH219A9

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Status:

US Approved Rx (2021)

First approved in 2021

Class (Stereo):

CHEMICAL (ABSOLUTE)

Targets:

Molybdopterin synthase catalytic subunit

Conditions:

molybdenum cofactor deficiency Type A

Fosdenopterin (NulibryTM) is a synthetic cyclic pyranopterin monophosphate that is being developed by Origin Biosciences (a subsidiary of BridgeBio Pharma) for the treatment of molybdenum cofactor deficiency (MoCD) type A. Patients with MoCD Type A h...

ave mutations in the MOCS1 gene leading to deficient MOCS1A/B dependent synthesis of the intermediate substrate, cPMP. Substrate replacement therapy with NULIBRY provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase (SOX), an enzyme that reduces levels of neurotoxic sulfites. Fosdenopterin was approved by the US FDA in February 2021 for use in reducing the risk of mortality in paediatric and adult patients with MoCD type A.

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