Cyclerion Gets Orphan Drug Designation for Zagociguat for Mitochondrial Diseases

 Cyclerion Therapeutics, Inc. (Nasdaq: CYCN), a clinical-stage biopharmaceutical company on a mission to develop treatments for serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to zagociguat (previously CY6463) for the treatment of mitochondrial diseases.

Zagociguat is the first CNS-penetrant sGC stimulator to be developed as a symptomatic and potentially disease-modifying therapy for serious diseases that involve the CNS. In an open-label, 29-day study in patients with MELAS*, zagociguat treatment was associated with improvements in multiple disease-relevant biomarkers: mitochondrial function, inflammation, cerebral blood flow, functional brain connectivity, and visually evoked brain activation. These data coupled with data from preclinical studies in cells from mitochondrial disease patients and in zebrafish disease models support the potential of zagociguat as a treatment for MELAS/mitochondrial diseases.

https://www.biospace.com/article/releases/cyclerion-therapeutics-receives-u-s-fda-orphan-drug-designation-for-zagociguat-for-the-treatment-of-mitochondrial-diseases/