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-[[(2S)-2-[[(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, sold under the brand name Forzinity, is a medication used for the treatment of Barth syndrome. [1] 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. [2]

Elamipretide was approved for medical use in the United States in September 2025. [2]

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). [2] Barth syndrome primarily affects males, typically starts with severe heart failure in infancy, and causes premature death. [2] People who survive into adolescence and adulthood often have fatigue, poor stamina, and exercise intolerance. [2] The quality of life and daily functioning of people with Barth syndrome are significantly affected throughout their lives. [2]

Society and culture

Elamipretide was approved for medical use in the United States in September 2025. [2] The US Food and Drug Administration (FDA) granted the application for elamipretide priority review and rare pediatric disease designations. [2] The FDA granted accelerated approval of Forzinity to Stealth Biotherapeutics. [2]

Names

Elamipretide is the international nonproprietary name. [3]

Elamipretide is sold under the brand name Forzinity. [4]

References

  1. 1 2 3 4 5 6 https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/215244s000lbl.pdf
  2. 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 .
  3. ↑ World Health Organization (2016). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 75". WHO Drug Information. 30 (1). hdl: 10665/331046 .
  4. ↑ "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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