Cyclerion Therapeutics Receives U.S. FDA Orphan Drug Designation

Cyclerion Therapeutics (Nasdaq: CYCN) has received Orphan Drug Designation from the FDA for zagociguat for the treatment of mitochondrial diseases.

Cyclerion says the decision underscores the FDA’s recognition of zagociguat’s potential promise as a first-ever therapy for patients with mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), a rare, genetic disease.

The company’s share price rose nearly 30% following the news.


  • In an open-label, 29-day study in patients with MELAS, zagociguat treatment was associated with improvements in mitochondrial function, inflammation, cerebral blood flow, functional brain connectivity, and visually evoked brain activation.
  • MELAS is a complex orphan disease with no approved therapies. It can affect any organ, including the central nervous system (CNS) and symptoms include intense fatigue, muscle weakness, and pain, stroke-like episodes, encephalomyopathy, seizures, and headaches.
  • 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.
  • 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 Cyclerion Therapeutics

Cyclerion Therapeutics is a clinical-stage biopharmaceutical company developing treatments for serious diseases. Cyclerion’s portfolio includes novel sGC stimulators that modulate a key node in a fundamental signaling network in both the CNS and the periphery. The company is advancing a number of candidates including zagociguat for mitochondrial disease, CY3018 for the treatment of neuropsychiatric diseases and disorders, praliciguat for rare kidney disease and olinciguat, which the Company intends to out-license for cardiovascular diseases.

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Cyclerion Therapeutics Receives U.S. FDA Orphan Drug Designation

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