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Current FDA-Approved Treatments for Mitochondrial Disease
There are now FDA-approved treatments for some mitochondrialRelated to the mitochondria. diseases on the market.
It’s an exciting time for the mitochondrial disease community, as pharmaceutical companies are bringing new FDA-approved treatments for mito to market. There are also several promising treatments and therapies currently undergoing clinical trials. As the FDA approves new treatment options for mito, we will update this page.
The following drugs and therapies have now been approved by the Food and Drug Administration (FDA) for the treatment of mitochondrial diseases:
KYGEVVI: An FDA-Approved Treatment for TK2D
In November 2025, the U.S. Food and Drug Administration (FDA) approved KYGEVVI to treat TK2D in adults and pediatric patients who started to show symptoms when they were 12 years old or younger. People with TK2D can’t properly make or maintain the DNA inside their muscle cells’ mitochondria. KYGEVVI contains two lab-made building blocks of DNA (pyrimidine nucleosides) called doxecitine and doxribtimine). When a person takes KYGEVVI, these DNA building blocks are taken up by their muscle cells and incorporated into the mitochondrial DNA. This helps restore the number of mitochondrial DNA copies in a cell, which is critical for muscle function. Learn more about TK2D—including treatment—on our Disease Fact Sheet page.
FORZINITY: An FDA-Approved Treatment for Barth Syndrome
In September 2025, the U.S. Food and Drug Administration (FDA) approved the first treatment for Barth syndromeA rare, genetic disorder of lipid metabolism that primarily affects males.. FORZINITY (also known as elamipretide), is approved for people with Barth syndrome who weigh at least 30kg (just over 66 pounds). Mitochondria need a special fat called cardiolipin to maintain their structure, stay organized, and work efficiently. In Barth syndrome, the damaged protein results in poor-quality (immature) cardiolipin. This lack of support means the mitochondria struggle to produce enough energy, leading to symptoms like weak muscles. Forzinity is designed to target the mitochondria, where it binds to cardiolipin. Forzinity helps the mitochondria create more energy, which in turn can lead to improved muscle strength and function. Learn more about Barth Syndrome—including treatment—on our Disease Fact Sheet page.
