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
Next:
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
Previous
Next:
Images
No images available for this drug.
Previous
Next:
Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.
