Mitotech Granted Orphan Drug Designation by FDA for Visomitin in LHON

Mitotech S.A, a Luxembourg-based clinical-stage biotechnology company in ophthalmology, announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) for the company’s topical cardiolipin peroxidation inhibitor, Visomitin, for the treatment of Leber’s Hereditary Optic Neuropathy (LHON), a rare inherited condition that can lead to blindness. Mitotech is planning to start a Phase 2 with Visomitin in LHON in 2022 in collaboration with the LHON clinic at the Doheny Eye Institute at the University of California Los Angeles (UCLA).

Visomitin demonstrated consistent improvement in visual acuity over several years in LHON patients involved in a three-year open-label Phase 2a study conducted outside the United States. Improvements were seen in patients with a range of underlying mutations, including those with variants such as G11778A where the chances of improvement are low. Mitotech’s planned Phase 2 study aims to develop Visomitin as a convenient and potentially high impact treatment for LHON.

“The US Orphan Drug Designation is highly encouraging for our efforts to bring Visomitin to young people whose lives have been radically affected by LHON,” said Natalia Perekhvatova, Chief Executive Officer of Mitotech S.A. “Visomitin has an outstanding safety profile and our preliminary human studies have demonstrated effectiveness in LHON. We are looking forward to progressing the drug in LHON as part of our wider ophthalmology pipeline.”

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