Zynerba Pharmaceuticals (NASDAQ:ZYNE) Inc announced it had received orphan drug designation for Zygel from the European Commission.
Zygel is the first and only pharmaceutically-manufactured cannabidiol formulated as a patent-protected permeation-enhanced clear gel, designed to provide controlled drug delivery into the bloodstream transdermally.
Devon, Pennsylvania-based Zynerba is developing Zygel to treat Fragile X Syndrome (FXS), a rare genetic developmental disability that is the leading known cause of both inherited intellectual disability and autism spectrum disorder.
READ: Zynerba Pharmaceuticals eyeing topline data from 22q Zygel trial mid-year as it completes patient enrollment
Zygel already has orphan drug designation in the US. The designation is given to medicines that treat, prevent or diagnose a rare life-threatening or debilitating disease.
Thanks to the orphan drug status, Zygel may now receive 10-year exclusivity in the European Union (EU) following full regulatory approval and reductions in EMA application fees, as well as access to protocol assistance.
Zynerba said it believes there are approximately 105,000 patients with FXS in the European Union, or 121,000 if the UK is included.
"Orphan drug designation by the EC is another important milestone for Zygel," said Armando Anido, CEO of Zynerba said in a statement. "It underscores the urgent, unmet medical need for patients diagnosed with Fragile X syndrome, and will benefit us as we continue to advance our clinical programs and prepare to bring Zygel to market."
The firm is currently enrolling patients in RECONNECT, a confirmatory pivotal Phase 3 trial of Zygel in children and adolescents with FXS, and continues to expect topline results from this trial in the second half of 2023.
Zynerba believes that the successful completion of the current development program for Zygel in FXS will satisfy the requirements of a marketing authorization application in the EU.
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