Zynerba Pharmaceuticals, Inc. announced that the European Commission (EC) has granted orphan drug designation to cannabidiol, the active ingredient in its transdermal gel, Zygel™, for the treatment of 22q11.2 deletion syndrome (22q). Orphan drug designation is granted to medicines that treat, prevent or diagnose a life-threatening or chronically debilitating rare disease, with a prevalence in the European Union (EU) of not more than 5 in 10,000, and with either no currently approved method of diagnosis, prevention or treatment or with significant benefit to those affected by the disease. The designation provides certain benefits to Zynerba, including 10-year EU market exclusivity upon regulatory approval, if received, reductions in European Medicines Agency application fees, and access to protocol assistance.

The Company believes there are approximately 112,000 patients with 22q in the EU and approximately 129,000 in Europe including the United Kingdom. Zygel was previously granted orphan drug designation by the EC for the treatment of Fragile X syndrome (FXS) and by U.S. Food and Drug Administration (FDA) for the treatment of FXS and 22q. Additionally, the FDA has granted Fast Track designation for Zygel for the treatment of behavioral symptoms associated with FXS.

In June 2022, the Company announced positive topline results from the exploratory, open label Phase 2 INSPIRE trial of Zygel in children and adolescents with 22q (Press Release). Based on the positive Phase 2 data, the Company requested and has been 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.