Regulatory Breakthrough for Potential Freidreich's Ataxia Treatment

CT-1601, the lead investigational drug candidate of Chondrial Therapeutics, Inc., has been granted Orphan Drug Desgination by the U.S. Food and Drug Administration (FDA) for the treatment of Freidreich’s Ataxia.

In February, the budding biotechnology company licensed the investigational compound, and announced that it had secured $22.6 million in financing from Deerfield Management to advance the treatment.

Friedreich’s ataxia is a hereditary condition in which issues pertaining to movement and functions throughout the body are the result of damage to the nervous system. It most commonly begins in patients between 5 and 18 years old, and leads to a gradual impairment and total loss of muscle coordination.

The degenerative neuromuscular disease currently affects an approximated 5,000 people in the U.S., and an estimated 10,000 across Europe.

Through CTI-1601, a carrier protein delivers frataxin, the deficient protein in the condition, to the mitochondria. There, researchers believe it is processed to develop the protein and becomes active in mitochondrial metabolism. It is believed that by replacing the deficient protein, the mitochondria will resume normal function, and symptoms in patients could be significantly reduced, and disease progression could be shortened.

"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,” said Carole Ben-Maimon, MD, president and CEO of Chondrial Therapeutics via press release.

Chondrial, who is focused on the treatment of rare mitochondrial diseases, anticipates filing an Investigational New Drug (IND) application for CTI-1601 with the FDA. Upon acceptance of the application, Phase 1 clinical trials of the compound can be initiated.

"The progress of CTI-1601 from discovery to its licensing to Chondrial is an important milestone in the collective effort to advance this novel and pro
mising therapeutic approach to address the high unmet need for treatment for individuals with Friedreich’s Ataxia.,” Jennifer Farmer, Executive Director, Friedreich’s Ataxia Research Alliance (FARA) said in February.

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