Cyron Therapeutics' Duchenne Muscular Dystrophy (DMD) treatment candidate CYR-2001 has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA).
CYR-2001 is an innovative drug utilizing gene therapy technology, with a mechanism that fundamentally blocks disease progression by restoring dystrophin protein production in muscle cells. Preclinical trials showed over 3 times the efficacy of existing treatments, with long-lasting effects confirmed from a single dose.
With orphan drug designation, Cyron Therapeutics will receive various incentives including 7 years of market exclusivity, tax credit benefits, and priority consultation opportunities with the FDA. The company can also participate in expedited review programs, significantly shortening the approval process.
With approximately 300,000 patients suffering worldwide and no fundamental treatment available for DMD, successful development of CYR-2001 is expected to bring new hope to patients and families.