Elamipretide

Last updated

Elamipretide
Elamipretide structure.svg
Clinical data
Trade names Forzinity
Other namesSS-31, MTP-131, Bendavia
License data
Routes of
administration 		Subcutaneous
ATC code
  • None
Legal status
Legal status
Identifiers
  • (2S)-6-Amino-2-{{#parsoidfragment:6}}(2S)-2-{{#parsoidfragment:7}}(2R)-2-amino-5-(diaminomethylideneamino)pentanoyl]amino]-3-(4-hydroxy-2,6-dimethylphenyl)propanoyl]amino]-N-[(2S)-1-amino-1-oxo-3-phenylpropan-2-yl]hexanamide
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
CompTox Dashboard (EPA)
Chemical and physical data
Formula C32H49N9O5
Molar mass 639.802 g·mol−1
3D model (JSmol)
  • CC1=CC(O)=CC(C)=C1C[C@H](NC(=O)[C@H](N)CCCNC(N)=N)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CC2=CC=CC=C2)C(N)=O
  • InChI=1S/C32H49N9O5/c1-19-15-22(42)16-20(2)23(19)18-27(41-29(44)24(34)11-8-14-38-32(36)37)31(46)39-25(12-6-7-13-33)30(45)40-26(28(35)43)17-21-9-4-3-5-10-21/h3-5,9-10,15-16,24-27,42H,6-8,11-14,17-18,33-34H2,1-2H3,(H2,35,43)(H,39,46)(H,40,45)(H,41,44)(H4,36,37,38)/t24-,25+,26+,27+/m1/s1
  • Key:SFVLTCAESLKEHH-WKAQUBQDSA-N

Elamipretide (development codes SS-31, MTP-131), [2] sold under the brand name Forzinity, is a medication used for the treatment of Barth syndrome. [1] [3] Elamipretide is a mitochondrial cardiolipin binder. [1] Is is used as the hydrochloride salt. [1] It is given by injection under the skin (subcutaneous). [1]

Contents

The most common side effects identified in clinical trials include mild-to-moderate injection site reactions. [4]

Elamipretide was approved for medical use in the United States in September 2025. [4]

Medical uses

Elamipretide is indicated to improve muscle strength in people with Barth syndrome weighing at least 30 kilograms (66 lb). [1]

Barth syndrome is a rare, serious and life-threatening disease of the mitochondria (the energy-producing parts of cells). [4] Barth syndrome primarily affects males, typically starts with severe heart failure in infancy, and causes premature death. [4] People who survive into adolescence and adulthood often have fatigue, poor stamina, and exercise intolerance. [4] The quality of life and daily functioning of people with Barth syndrome are significantly affected throughout their lives. [4]

Elamipretide has also been researched for numerous other indications including heart failure, macular degeneration, enhanced wound healing and hypertension, though it is currently only medically approved for use in the treatment of Barth syndrome. [5] [6] [7] [8] [9] [10] [11] [12] [13] [14] [ excessive citations ]

Society and culture

Elamipretide was approved for medical use in the United States in September 2025. [4] The US Food and Drug Administration (FDA) granted the application for elamipretide priority review and rare pediatric disease designations. [4] The FDA granted accelerated approval of Forzinity to Stealth Biotherapeutics. [4]

Names

Elamipretide is the international nonproprietary name. [15]

Elamipretide is sold under the brand name Forzinity. [16]

References

  1. 1 2 3 4 5 6 https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/215244s000lbl.pdf [ bare URL PDF ]
  2. ↑ Szeto HH (2014). "First-in-class cardiolipin-protective compound as a therapeutic agent to restore mitochondrial bioenergetics". British Journal of Pharmacology. 171 (8): 2029–2050. doi:10.1111/bph.12461 (inactive 18 November 2025). PMC   3976620 . PMID   24117165.{{cite journal}}: CS1 maint: DOI inactive as of November 2025 (link)
  3. ↑ Jacob N, Schecter D, Marshall M, Bansal N, Lamour J, Vernon H, et al. (2025). "Elamipretide in the Management of Barth Syndrome: Current Evidence and a Case Report". Molecular Genetics and Metabolism. 146 (1–2) 109220. doi:10.1016/j.ymgme.2025.109220 (inactive 18 November 2025). PMID   40816230.{{cite journal}}: CS1 maint: DOI inactive as of November 2025 (link)
  4. 1 2 3 4 5 6 7 8 9 "FDA Grants Accelerated Approval to First Treatment for Barth Syndrome". U.S. Food and Drug Administration (FDA). 19 September 2025. Retrieved 20 September 2025.PD-icon.svg This article incorporates text from this source, which is in the public domain .
  5. ↑ Steggall A, Mordi I, Lang C (2017). "Targeting Metabolic Modulation and Mitochondrial Dysfunction in the Treatment of Heart Failure". Diseases. 5 (2): 14. doi: 10.3390/diseases5020014 (inactive 18 November 2025). PMC   5547981 . PMID   28933367.{{cite journal}}: CS1 maint: DOI inactive as of November 2025 (link)
  6. ↑ Cano Sanchez M, Lancel S, Boulanger E, Neviere R (2018). "Targeting Oxidative Stress and Mitochondrial Dysfunction in the Treatment of Impaired Wound Healing: A Systematic Review". Antioxidants. 7 (8): 98. doi: 10.3390/antiox7080098 (inactive 18 November 2025). PMC   6115926 . PMID   30042332.{{cite journal}}: CS1 maint: DOI inactive as of November 2025 (link)
  7. ↑ Eirin A, Lerman A, Lerman LO (2018). "Enhancing Mitochondrial Health to Treat Hypertension". Current Hypertension Reports. 20 (10) 89. doi:10.1007/s11906-018-0889-4 (inactive 18 November 2025). PMC   6524134 . PMID   30120623.{{cite journal}}: CS1 maint: DOI inactive as of November 2025 (link)
  8. ↑ Szeto HH (2019). "Stealth Peptides Target Cellular Powerhouses to Fight Rare and Common Age-Related Diseases". Protein & Peptide Letters. 25 (12): 1108–1123. doi:10.2174/0929866525666181101105209 (inactive 18 November 2025). PMID   30381054.{{cite journal}}: CS1 maint: DOI inactive as of November 2025 (link)
  9. ↑ Obi C, Smith AT, Hughes GJ, Adeboye AA (2022). "Targeting mitochondrial dysfunction with elamipretide". Heart Failure Reviews. 27 (5): 1925–1932. doi:10.1007/s10741-021-10199-2 (inactive 18 November 2025). PMID   35037146.{{cite journal}}: CS1 maint: DOI inactive as of November 2025 (link)
  10. ↑ Nhu NT, Xiao S, Liu Y, Kumar VB, Cui Z, Lee S (2022). "Neuroprotective Effects of a Small Mitochondrially-Targeted Tetrapeptide Elamipretide in Neurodegeneration". Frontiers in Integrative Neuroscience. 15 747901. doi: 10.3389/fnint.2021.747901 (inactive 18 November 2025). PMC   8801496 . PMID   35111001.{{cite journal}}: CS1 maint: DOI inactive as of November 2025 (link)
  11. ↑ Tung C, Varzideh F, Farroni E, Mone P, Kansakar U, Jankauskas SS, et al. (2025). "Elamipretide: A Review of Its Structure, Mechanism of Action, and Therapeutic Potential". International Journal of Molecular Sciences. 26 (3): 944. doi: 10.3390/ijms26030944 (inactive 18 November 2025). PMC   11816484 . PMID   39940712.{{cite journal}}: CS1 maint: DOI inactive as of November 2025 (link)
  12. ↑ Sabbah HN, Alder NN, Sparagna GC, Bruce JE, Stauffer BL, Chao LH, et al. (2025). "Contemporary insights into elamipretide's mitochondrial mechanism of action and therapeutic effects". Biomedicine & Pharmacotherapy. 187 118056. doi:10.1016/j.biopha.2025.118056 (inactive 18 November 2025). PMC   12164653 . PMID   40294492.{{cite journal}}: CS1 maint: DOI inactive as of November 2025 (link)
  13. ↑ Radovic M, Gartzke LP, Wink SE, Van Der Kleij JA, Politiek FA, Krenning G (2025). "Targeting the Electron Transport System for Enhanced Longevity". Biomolecules. 15 (5): 614. doi: 10.3390/biom15050614 (inactive 18 November 2025). PMC   12109555 . PMID   40427507.{{cite journal}}: CS1 maint: DOI inactive as of November 2025 (link)
  14. ↑ Patel PS, Pabla NS, Bajwa A (2025). "Therapeutic Approaches Involving Mitochondria in the Treatment of Acute Kidney Injury". Seminars in Nephrology 151676. doi:10.1016/j.semnephrol.2025.151676 (inactive 18 November 2025). PMID   41027799.{{cite journal}}: CS1 maint: DOI inactive as of November 2025 (link)
  15. ↑ World Health Organization (2016). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 75". WHO Drug Information. 30 (1). hdl: 10665/331046 .
  16. ↑ "Stealth BioTherapeutics Announces FDA Accelerated Approval of Forzinity (elamipretide HCl), the First Therapy for Progressive and Life-limiting Ultra-rare Genetic Disease Barth Syndrome" (Press release). Stealth Biotherapeutics. 19 September 2025. Retrieved 20 September 2025– via PR Newswire.

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