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 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."

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.

About Mitotech S.A.

Mitotech S.A. is a Luxembourg-based late clinical stage biotechnology company developing drugs to treat the mitochondrial oxidative stress underlying predominantly age-related ophthalmology disorders. Mitotech’s products are based on novel small molecules that inhibit cardiolipin peroxidation within mitochondria. For more information, please visit www.mitotechpharma.com.

About Visomitin

Visomitin is a cardiolipin peroxidation inhibitor, under development for multiple indications including LHON, dry eye disease (DED) and glaucoma. Visomitin was designed to sustain and restore mitochondrial function and interrupt apoptosis in mitochondrial conditions like LHON. In DED Visomitin’s novel multimodal action targets inflammation, corneal/conjunctival damage, tear deficiency, and gland tissue regeneration. Visomitin has previously completed two late-stage DED trials (VISTA 1/2) in which it showed significant staining clearance in a major dry-eye subset, whilst also exhibiting placebo-like tolerability.

About LHON

Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease that mainly affects adolescents and young adults. LHON is characterized by the degeneration of retinal ganglion cells resulting in irreversible vision loss that can lead to blindness. LHON is associated with painless, sudden loss of central vision starting in one eye, and then progressing to the second eye. Ultimately, it is a symmetrical disease with poor functional visual recovery. Approximately 800-1,200 new patients lose their sight each year in the United States and the European Union as a result of LHON.

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