The Food and Drug Administration (FDA) has granted Orphan Drug designation to visomitin for the treatment of Leber hereditary optic neuropathy (LHON), a rare genetic disease characterized by the degeneration of retinal ganglion cells that can result in irreversible vision loss.

Visomitin is a cardiolipin peroxidation inhibitor designed to sustain and restore mitochondrial function and interrupt apoptosis in mitochondrial conditions such as LHON. The designation is supported by data from a 3-year, open-label phase 2a study conducted outside the United States.

Results from the study showed that treatment with visomitin was associated with consistent improvement in visual acuity in LHON patients. Improvements were seen in patients with a range of underlying mutations, including those with variants such as G11778A.

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“Visomitin has an outstanding safety profile and our preliminary human studies have demonstrated effectiveness in LHON,” said Natalia Perekhvatova, Chief Executive Officer of Mitotech S.A. “We are looking forward to progressing the drug in LHON as part of our wider ophthalmology pipeline.”

A phase 2 study evaluating visomitin in LHON patients is expected to begin in 2022.

The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals. 


Mitotech granted Orphan Drug designation by FDA for Visomitin in LHON. News release. Mitotech. Accessed December 16, 2021.

This article originally appeared on MPR