Fosmetpantotenate

Names

[ Name ]:
Fosmetpantotenate

Biological Activity

[Description]:

Fosmetpantotenate is a phosphopantothenic acid (PPA) prodrug designed to release PPA intracellularly, leading to restoration of CoA levels. Fosmetpantotenate is used to treat pantothenate kinase-associated neurodegeneration.

[Related Catalog]:

Research Areas >> Neurological Disease

[References]

[1]. Elbaum D, Beconi MG, Monteagudo E, et al. Fosmetpantotenate (RE-024), a phosphopantothenate replacement therapy for pantothenate kinase-associated neurodegeneration: Mechanism of action and efficacy in nonclinical models. PLoS One. 2018;13(3):e0192028. Published 2018 Mar 9.

[2]. Klopstock T, Escolar ML, Marshall RD, et al. The FOsmetpantotenate Replacement Therapy (FORT) randomized, double-blind, Placebo-controlled pivotal trial: Study design and development methodology of a novel primary efficacy outcome in patients with pantothenate kinase-associated neurodegeneration. Clin Trials. 2019;16(4):410-418.

Chemical & Physical Properties

[ Molecular Formula ]:
C₂₀H₃₁N₂O₉P

[ Molecular Weight ]:
474.44

Related Compounds

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