Primary mitochondrial myopathy and bioenergetic disorders — compounds assessed

§4Compounds assessed

Compounds the Institute assesses in this indication, with the class of each and its overall certainty. A compound appears here whether or not the evidence supports its use, because recording that a compound has been studied and found wanting is as much part of the assessment as recording that one has not.

• SS-31 (elamipretide) in mitochondrial disease

  Mitochondria-targeted cardiolipin-binding tetrapeptide2 contributing trialsfull monograph

  A phase 3 trial in primary mitochondrial myopathy did not meet its primary endpoints of six-minute walk distance and symptom score at 24 weeks — The Institute rates the negative finding moderate certainty. A well-conducted phase 3 trial with a clear null…

  Moderate
• MOTS-c in mitochondrial disease

  Mitochondrial-derived 16-residue peptide0 contributing trialsfull monograph

  No randomised controlled human trial identified — —

  Very low
• NAD+ (nicotinamide adenine dinucleotide) in mitochondrial disease

  Endogenous pyridine dinucleotide coenzyme0 contributing trialsfull monograph

  No assessable randomised evidence identified for administered NAD+ — —

  Very low

References cited on this page

References are numbered in order of first citation in this document. Each superscript in the text links to its entry below.

1. Szeto HH. First-in-class cardiolipin-protective compound as a therapeutic agent to restore mitochondrial bioenergetics. British Journal of Pharmacology 2014;171(8):2029–2050. doi:10.1111/bph.12461 · PMID 24117165

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