Primary mitochondrial myopathy and bioenergetic disorders — evidence across compounds
Every compound the Institute assesses in primary mitochondrial myopathy and bioenergetic disorders, with its certainty rating.
§2Evidence across compounds
Every compound the Institute assesses in this indication, with the effect as recorded, the certainty rating and the contributing trials. Effects are reported on the anchor outcome where the contributing trials measured it and on the outcome the trial actually reported where they did not.
Table 1. Compounds assessed in primary mitochondrial myopathy and bioenergetic disorders, ordered by certainty.
| Compound | Effect as recorded | Interval as reported | Certainty | Trials |
|---|---|---|---|---|
| SS-31 (elamipretide)Mitochondria-targeted cardiolipin-binding tetrapeptide | 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 | negative result on both primary endpoints | Moderate | 2 |
| MOTS-cMitochondrial-derived 16-residue peptide | No randomised controlled human trial identified | — | Very low | 0 |
| NAD+ (nicotinamide adenine dinucleotide)Endogenous pyridine dinucleotide coenzyme | No assessable randomised evidence identified for administered NAD+ | — | Very low | 0 |
| Estimates in this table are not on a common scale and must not be subtracted from one another. Where a comparison between two compounds has been made directly, it appears in a synthesis and not here. | ||||
§2.1Syntheses bearing on this indication
- Mitochondria-targeted peptides in bioenergetic disorders — Low
- SS-31 (elamipretide) in primary mitochondrial myopathy and bioenergetic disorders: effect on the anchor outcome — Moderate
- SS-31 (elamipretide) in primary mitochondrial myopathy and bioenergetic disorders: tolerability and discontinuation — Moderate
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.
- 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
- D’Hondt M, Bracke N, Taevernier L, Gevaert B, Verbeke F, Wynendaele E, De Spiegeleer B. Related impurities in peptide medicines. Journal of Pharmaceutical and Biomedical Analysis 2014;101:2–30. doi:10.1016/j.jpba.2014.06.012 · PMID 25044089
Identifiers are reproduced only where the Institute holds them. Where a digital object identifier or PubMed identifier is not shown, the Institute has recorded the journal and year and has not constructed an identifier.