Elamipretide
Chemical compound
From Wikipedia, the free encyclopedia
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] It is used as the hydrochloride salt.[1] It is given by injection under the skin (subcutaneous).[1]
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| Trade names | Forzinity |
| Other names | SS-31, MTP-131, Bendavia |
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| Routes of administration | Subcutaneous |
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| Formula | C32H49N9O5 |
| Molar mass | 639.802 g·mol−1 |
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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
Legal status
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]
