Cyclerion Gets FDA Orphan Designation for Zagociguat in Mitochondrial Diseases

By Colin Kellaher

Cyclerion Therapeutics Inc. on Monday said the U.S. Food and Drug Administration granted orphan-drug designation to its lead drug candidate zagociguat for the treatment of mitochondrial diseases, a group of genetic disorders.

The Cambridge, Mass., clinical-stage biopharmaceutical company said zagociguat holds promise as a first-ever therapy for patients with mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes, or MELAS, a mitochondrial disease that mainly affects the nervous system and muscles.

The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S. and provides for an extended marketing exclusivity period against competition.

Write to Colin Kellaher at [e-mail]

(END) Dow Jones Newswires

03-27-23 0736ET