The Food and Drug Administration (FDA) has granted Orphan Drug designation to VTA-110 (Vita Therapeutics) for the treatment of Duchenne muscular dystrophy (DMD).

VTA-110 is a potential first-in-class allogenic induced pluripotent stem cell (iPSC)-based therapy that has demonstrated the ability to repair and regenerate healthy muscle in preclinical studies. The Company expects to progress the investigational drug into clinical trials as well as further investigate the product for other congenital muscular dystrophy diseases.

Douglas Falk, MS, CEO of Vita Therapeutics commented, “We are very pleased the FDA granted Orphan Drug designation for VTA-110. We believe VTA-110 has the potential to be a long-term disease-modifying treatment for patients living with DMD and other types of muscular dystrophy.”   

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Orphan drug designation is granted to medicine intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals.

Additional information on Vita Therapeutics can be found here.