Chondrial Therapeutics Inc. has been granted orphan drug status by the U.S. Food and Drug Administration for CTI-1601, its drug candidate to treat Friedreich’s ataxia.
The Pennsylvania-based biotechnology company licenses intellectual property developed at the Indiana University School of Medicine. This IP was invented by Dr. R. Mark Payne, professor of pediatric cardiology at the IU School of Medicine and director of the Translational Research Training Program of the Indiana Clinical Translational Sciences Institute.
Orphan drug status is designated for drugs that provide treatment of diseases that affect less than 200,000 people in the United States.
Dr. Carole Ben-Maimon, president and CEO of Chondrial Therapeutics, said, “We are very pleased to receive orphan drug designation for CTI-1601. Our team is working to complete all Investigational New Drug enabling studies so that we can file an IND and begin human clinical trials.”
More information about Chondrial Therapeutics, Friedreich’s ataxia and the U.S. FDA orphan drug status is available online.