Luxembourg-based Mitotech SA announced that the 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 cause blindness.
In 2022, Mitotech plans to begin a phase 2 study with Visomitin in LHON in partnership with the LHON clinic at the University of California Los Angeles' Doheny Eye Institute (UCLA).
Visomitin demonstrated consistent improvement in visual acuity over several years in LHON patients involved in a 3-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, Chief Executive Officer of Mitotech, said in a company news release.
"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's Office of Orphan Products Development (OOPD) promotes the development and testing of novel medicines for diseases that affect fewer than 200,000 persons in the United States.
Orphan drug status confers a number of advantages, including seven years of market exclusivity upon regulatory approval, waiver of FDA application fees, and tax incentives for eligible clinical trials.
Leber hereditary optic neuropathy (LHON) is an inherited form of vision loss. Although this condition usually begins in a person's teens or twenties, rare cases may appear in early childhood or later in adulthood. For unknown reasons, males are affected much more often than females.
Blurring and clouding of vision are usually the first symptoms of LHON. These vision problems may begin in one eye or simultaneously in both eyes; if vision loss starts in one eye, the other eye is usually affected within several weeks or months. Over time, vision in both eyes worsens with a severe loss of sharpness (visual acuity) and color vision.
Mutations in the MT-ND1, MT-ND4, MT-ND4L, or MT-ND6 gene can cause LHON. These genes are found in the DNA of cellular structures called mitochondria, which convert the energy from food into a form that cells can use.
Leber hereditary optic neuropathy currently has no proven treatment. Symptomatic treatment and genetic counseling are important in the management of patients with Leber hereditary optic neuropathy.