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Mitotech granted Orphan Drug Designation by FDA for Visomitin in LHON


The US Orphan Drug Designation would provide seven-year market exclusivity if Visomitin was approved for use in LHON.

Mitotech SA, announced today 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.a Luxembourg-based clinical-stage biotechnology company in ophthalmology, 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).

According to the company, 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," Natalia Perekhvatova, CEO of Mitotech, said in a statement. "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."

The FDA Office of Orphan Products Development (OOPD) supports and advances the development and evaluation of new treatments for rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain benefits, including seven-year market exclusivity upon regulatory approval if received, exemption from FDA application fees and tax credits for qualified clinical trials.

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