SS-31 (elamipretide) in primary mitochondrial myopathy and bioenergetic disorders — evidence extract
The Institute's graded assessment of SS-31 (elamipretide) for primary mitochondrial myopathy and bioenergetic disorders, with the contributing trials and the domain-by-domain certainty reasoning.
§1Evidence extract: Primary mitochondrial myopathy and bioenergetic disorders
§1.1Question and anchor outcome
- Population
- Genetically determined impairment of oxidative phosphorylation producing myopathy, exercise intolerance or multisystem disease.
- Intervention
- SS-31 (elamipretide), subcutaneous or intravenous in clinical studies
- Comparator
- As used in each contributing trial; reported per trial rather than pooled across comparator types
- Anchor outcome
- Six-minute walk distance
Additional outcomes the Institute extracts for this indication: Primary mitochondrial myopathy symptom assessment; Muscle phosphocreatine recovery kinetics.
§1.2Contributing trials
Table 1. Trials contributing to the assessment of SS-31 (elamipretide) in primary mitochondrial myopathy and bioenergetic disorders.
| Trial | Phase | Design | Randomised | Duration | Year |
|---|---|---|---|---|---|
| MMPOWER-3 | 3 | Randomised, double-blind, placebo-controlled | 218 | 24 weeks | 2022 |
| MMPOWER-2 | 2 | Randomised crossover | 30 | 4 weeks per period | 2018 |
§1.3Certainty assessment
Table 2. Reasoning recorded against each certainty domain.
| Domain | Rating | Reasoning |
|---|---|---|
| Risk of bias | No concern | No serious concern identified in this domain. |
| Inconsistency | No concern | No serious concern identified in this domain. |
| Indirectness | No concern | No serious concern identified in this domain. |
| Imprecision | No concern | No serious concern identified in this domain. |
| Publication bias | Serious | Too few contributing studies for a formal assessment; the risk cannot be excluded. |
| Overall rating: Moderate certainty. The true effect is likely to be close to the estimate, but there is a possibility that it is substantially different. Further research is likely to have an important impact on confidence in the estimate. | ||
§1.4What this extract does not establish
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
- Guyatt GH, Oxman AD, Kunz R, Brozek J, Alonso-Coello P, Rind D, Devereaux PJ, Montori VM, Freyschuss B, Vist G, Jaeschke R, Williams JW, Murad MH, Sinclair D, Falck-Ytter Y, Meerpohl J, Whittington C, Thorlund K, Andrews J, Schünemann HJ. GRADE guidelines: 6. Rating the quality of evidence — imprecision. Journal of Clinical Epidemiology 2011;64(12):1283–1293. doi:10.1016/j.jclinepi.2011.01.012 · PMID 21839614
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.