Cyclerion’s Mitochondrial Diseases Candidate Gets Orphan Drug Nod

The FDA has granted Orphan Drug designation to Cyclerion Therapeutics’ investigational drug zagociguat for treatment of mitochondrial diseases.

Zagociguat is targeted at several diseases that involve the central nervous system, including mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes syndrome, a condition that affects multiple organ systems including the central nervous system.

In a 29-day study, the drug candidate was associated with improvements in several disease-relevant biomarkers, including mitochondrial function, inflammation, cerebral blood flow, functional brain connectivity and visually evoked brain activation.

The FDA’s Orphan Drug program offers incentives such as tax credits for clinical testing, user fee exemptions and seven-year marketing exclusivity upon FDA approval. The program is for drugs intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the U.S.