elamipretide (Pending FDA Approval)

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Dosing & Uses

Primary Mitochondrial Diseases

Pending FDA approval for Barth syndrome and primary mitochondrial myopathy

Orphan Designations

Primary mitochondrial myopathy

Barth syndrome

Leber’s Hereditary Optic Neuropathy

Orphan sponsor

  • Stealth BioTherapeutics Inc; 275 Grove Street 3-107; Newton, Massachusetts 02466
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Pharmacology

Mechanism of Action

Aromatic-cationic tetrapeptide that penetrates cell membranes and localizes to the inner mitochondrial membrane where it associates with cardiolipin to improve mitochondrial cristae architecture

In primary mitochondrial diseases, dysfunctional mitochondria produce less ATP and increases unhealthy levels of reactive oxygen species (ROS), which leads to oxidative stress

Elamipretide improves energy production, reduces harmful oxidative stress by decreasing ROS production, and ultimately increases ATP supplied to affected cells and organs

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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.