Chondrial Announces FDA Orphan Drug Designation for CTI-1601, a Novel Investigational Technology for the Treatment of Friedreich’s Ataxia


BALA CYNWYD, Pa.Aug. 3, 2017 — Chondrial Therapeutics, Inc., an emerging biotechnology company focused on the treatment of rare mitochondrial diseases, announced today that the US Food and Drug Administration (FDA) has granted orphan drug designation to its lead investigational drug candidate, CTI-1601, being developed for the treatment of Friedreich’s Ataxia.


Orphan drug designation is granted by the FDA Office of Orphan Products Development to drugs being developed for safe and effective treatment of diseases that affect fewer than 200,000 people in the U.S.  For a drug to qualify for orphan drug designation, FDA must determine there is a medically plausible basis for the use of the drug for the rare disease or condition.


Orphan drug status makes the investigational drug eligible for certain development incentives, including tax credits for clinical testing and exemption from a prescription drug user fee. Orphan drug designation also may entitle the company to a period of seven years of market exclusivity upon FDA approval of the drug.


Carole Ben-Maimon, MD, president and CEO of Chondrial Therapeutics, commented, “We are very pleased to receive orphan drug designation for CTI-1601.  Our team is working to complete all Investigational New Drug (IND) enabling studies so that we can file an IND and begin human clinical trials.”


About Friedreich’s Ataxia


Friedreich’s Ataxia (FA) is a progressive disease that affects multiple body systems, particularly the brain and heart. FA is the most common ataxia in man and is caused by a deficiency of a key protein, frataxin, which is normally present in the mitochondria of all tissues.  Children and young adults progressively lose the ability to walk and speak, and their hearts become very thick and weak, and can eventually fail. There is currently no cure for FA.


About Chondrial Therapeutics


Chondrial Therapeutics is an emerging biotechnology company focused on the treatment of rare mitochondrial diseases. Chondrial’s lead compound is CTI-1601, which the Company is developing as a potential treatment for Friedreich’s Ataxia. CTI-1601 is designed to deliver the frataxin protein to the mitochondria of patients with Friedreich’s Ataxia, who, due to a genetic abnormality, are unable to produce this essential protein.