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
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 code SS-31), [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]

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. First-in-class cardiolipin-protective compound as a therapeutic agent to restore mitochondrial bioenergetics. Br J Pharmacol. 2014 Apr;171(8):2029-50. doi : 10.1111/bph.12461 PMID   24117165
  3. ↑ Jacob N, Schecter D, Marshall M, Bansal N, Lamour J, Vernon H, Morava E, Elsharkawi I. Elamipretide in the Management of Barth Syndrome: Current Evidence and a Case Report. Mol Genet Metab. 2025 Sep-Oct;146(1-2):109220. doi : 10.1016/j.ymgme.2025.109220 PMID   40816230
  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 IR, Lang CC. Targeting Metabolic Modulation and Mitochondrial Dysfunction in the Treatment of Heart Failure. Diseases. 2017 May 10;5(2):14. doi : 10.3390/diseases5020014 {{doi}}: unflagged free DOI (link) PMID   28933367
  6. ↑ Cano Sanchez M, Lancel S, Boulanger E, Neviere R. Targeting Oxidative Stress and Mitochondrial Dysfunction in the Treatment of Impaired Wound Healing: A Systematic Review. Antioxidants (Basel). 2018 Jul 24;7(8):98. doi : 10.3390/antiox7080098 {{doi}}: unflagged free DOI (link) PMID   30042332
  7. ↑ Eirin A, Lerman A, Lerman LO. Enhancing Mitochondrial Health to Treat Hypertension. Curr Hypertens Rep. 2018 Aug 17;20(10):89. doi : 10.1007/s11906-018-0889-4 PMID   30120623
  8. ↑ Szeto HH. Stealth Peptides Target Cellular Powerhouses to Fight Rare and Common Age-Related Diseases. Protein Pept Lett. 2018;25(12):1108-1123. doi : 10.2174/0929866525666181101105209 PMID   30381054
  9. ↑ Obi C, Smith AT, Hughes GJ, Adeboye AA. Targeting mitochondrial dysfunction with elamipretide. Heart Fail Rev. 2022 Sep;27(5):1925-1932. doi : 10.1007/s10741-021-10199-2 PMID   35037146
  10. ↑ Nhu NT, Xiao SY, Liu Y, Kumar VB, Cui ZY, Lee SD. Neuroprotective Effects of a Small Mitochondrially-Targeted Tetrapeptide Elamipretide in Neurodegeneration. Front Integr Neurosci. 2022 Jan 17;15:747901. doi : 10.3389/fnint.2021.747901 {{doi}}: unflagged free DOI (link) PMID   35111001
  11. ↑ Tung C, Varzideh F, Farroni E, Mone P, Kansakar U, Jankauskas SS, Santulli G. Elamipretide: A Review of Its Structure, Mechanism of Action, and Therapeutic Potential. Int J Mol Sci. 2025 Jan 23;26(3):944. doi : 10.3390/ijms26030944 {{doi}}: unflagged free DOI (link) PMID   39940712
  12. ↑ Sabbah HN, Alder NN, Sparagna GC, Bruce JE, Stauffer BL, Chao LH, Pitceathly RDS, Maack C, Marcinek DJ. Contemporary insights into elamipretide's mitochondrial mechanism of action and therapeutic effects. Biomed Pharmacother. 2025 Jun;187:118056. doi : 10.1016/j.biopha.2025.118056 PMID   40294492
  13. ↑ Radovic M, Gartzke LP, Wink SE, van der Kleij JA, Politiek FA, Krenning G. Targeting the Electron Transport System for Enhanced Longevity. Biomolecules. 2025 Apr 23;15(5):614. doi : 10.3390/biom15050614 {{doi}}: unflagged free DOI (link) PMID   40427507
  14. ↑ Patel PS, Pabla NS, Bajwa A. Therapeutic Approaches Involving Mitochondria in the Treatment of Acute Kidney Injury. Semin Nephrol. 2025 Sep 29:151676. doi : 10.1016/j.semnephrol.2025.151676 PMID   41027799
  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.

Further reading


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