Business
Cyclerion Therapeutics Receives U.S. FDA Orphan Drug Designation for Zagociguat for the Treatment of Mitochondrial Diseases
CAMBRIDGE, Mass., March 27, 2023 (GLOBE NEWSWIRE) -- Cyclerion Therapeutics, Inc. (Nasdaq: CYCN), a clinical-stage biopharmaceutical company on a mission to
About this update from Cyclerion Therapeutics, Inc.
[{"type":"text","content":"CAMBRIDGE, Mass., March 27, 2023 (GLOBE NEWSWIRE) -- 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. “Orphan drug designation underscores the FDA’s recognition of zagociguat’s potential promise as a first-ever therapy for patients with MELAS, a rare, genetic mitochondrial disease,” said Peter Hecht, Ph.D., Chief Executive Officer of Cyclerion. “Cyclerion is working expeditiously to advance this potential treatment to help address the immense unmet needs of patients with MELAS, a patient population in desperate need of therapies.” The FDA's Orphan Drug Designation program provides orphan status to drugs defined as those intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions, and seven-year marketing exclusivity upon FDA approval. About MELAS MELAS is a complex orphan disease affecting multiple organ systems, including the CNS, with different degrees of severity, and no approved therapies. MELAS, one of the most common primary mitochondrial diseases (PMDs), is caused by mitochondrial DNA mutations resulting in large clusters of familial cases. It is estimated that about 1 in 4,300 individuals has a mitochondrial disease, and ~80% of individuals with m...