Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) reported it has further advanced its transdermal gel Zygel—receiving orphan drug designation from the European Commission (EC) for the gel's active ingredient to treat 22q11.2 deletion syndrome (22q).
Zygel, which contains cannabidiol, was previously granted orphan drug designation by the EC for the treatment of Fragile X syndrome (FXS).
The US Food and Drug Administration (FDA) has granted it orphan drug status for the treatment of FXS and 22q. The US regulator has also granted Fast Track designation for Zygel for behavioral symptoms associated with FXS.
"We are pleased to have been granted orphan drug designation in the U.S. and now the EU, which we believe reflects the unmet medical need for new therapies to treat 22q," said Armando Anido, the CEO of Zynerba in a statement.
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"Orphan drug designation is another important step forward in advancing Zygel as a potential new treatment option for patients with 22q."
The second most common chromosomal disorder after Down syndrome, 22q is caused by a small missing piece of the 22nd chromosome.
Physical symptoms include characteristic palate abnormalities, heart defects, immune dysfunction, and esophageal / GI issues, as well as debilitating neuropsychiatric and behavioral challenges.
The company believes there are around 112,000 patients with 22q in the EU and approximately 129,000 in Europe, including the UK.
Orphan drug designation is granted to medicines that treat, prevent or diagnose a life-threatening or chronically debilitating rare disease.
In June this year, Zynerba Pharmaceuticals unveiled positive topline results from an exploratory, open label Phase 2 INSPIRE trial of Zygel in children and adolescents with 22q.
Based on this, the company was granted an initial meeting with the FDA before the end of 2022 to obtain feedback on the Phase 2 data and regulatory pathway for Zygel in patients with 22q.
Contact the writer at giles@proactiveinvestors.com