MitoChem Therapeutics Granted FDA Orphan Drug Designation for Treatment of Retinitis Pigmentosa

CHARLESTON, SC / ACCESSWIRE / April 14, 2021 / MitoChem Therapeutics, Inc., a privately held company developing therapies for diseases involving mitochondrial dysfunction, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation status to its lead compound, MC16, for treatment of retinitis pigmentosa (RP). Retinitis pigmentosa is an inherited degenerative disease of the retina caused by genetic mutations that lead to severe vision loss and blindness. There are currently no approved treatments for RP.

One of the key challenges of developing a treatment for RP is that it is not caused by a single mutation but is a composition of related diseases caused by hundreds of different genetic mutations. One of the hallmarks of retinal degeneration is mitochondrial dysfunction. Mitochondria, "the powerhouse of the cell," produce the vast majority of the energy our cells need to function and survive. Because the eye is one of the highest energy-consuming systems in the body, sustained disruption of mitochondrial function results in energy deficits and ultimately leads to cell death. Mitochondrial dysfunction is recognized as an underlying driver of pathology in retinal diseases.

"MC16 is unique in that it is designed to be a gene-mutation agnostic treatment with the potential to treat all forms of RP," said Dr. Bärbel Rohrer, PhD, Chief Science Officer and co-founder of MitoChem.

"Designation as an Orphan drug is an important step in advancing our therapies for retinitis pigmentosa and other retinal degeneration diseases." said Michael Voevodsky, Chief Executive Officer. "Everyone at MitoChem is driven to develop crucial treatments for patients with these devastating diseases."

The FDA's Orphan Drug Designation Program grants orphan status to drugs for the prevention, diagnosis, or treatment of diseases or conditions affecting fewer than 200,000 persons in the US. Orphan Drug Designation qualifies a candidate for various development incentives, including tax credits for eligible clinical trials, waiver of application fees, and market exclusivity for seven years upon FDA approval.

About MitoChem Therapeutic

MitoChem Therapeutics is a privately held company developing therapies for diseases involving mitochondrial dysfunction. The company was co-founded by the late Craig Beeson, PhD, and Bärbel Rohrer, PhD, to develop a new approach to treating neurodegeneration and aging diseases by focusing on protecting mitochondrial homeostasis. Early funding by Foundation Fighting Blindness and NIH has resulted in a proprietary library of mitochondrial protective compounds, and early proof of concept in numerous in vivo models for retinitis pigmentosa and age-related macular degeneration. The lead compound MC16 is a first-in-class small molecule that up regulates pro-survival and anti-oxidant pathways. The company is pursuing partnerships to develop non-ophthalmic indications such as Parkinson's, Alzheimer's, ALS, muscular dystrophies, diabetes and ischemia. For more information, please visit


Michael Voevodsky

SOURCE: MitoChem Therapeutics, Inc.


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