ZYNE - Zynerba rises on orphan drug status for cannabidiol Zygel for 22q11.2 deletion syndrome in EU

• The European Commission (EC) granted orphan drug designation to Zynerba Pharmaceuticals' ( NASDAQ: ZYNE ) cannabidiol, the active ingredient in the company's transdermal gel Zygel, to treat 22q11.2 deletion syndrome (22q).
• The 22q11.2 deletion syndrome is a disorder which occurs when a small part of chromosome 22 is missing. This deletion leads to development issues of several body systems.
• "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 Zynerba Chairman and CEO Armando Anido.
• The orphan drug status in the EU is aimed at therapies for treating or preventing diseases which affect fewer than five in 10,000 people in the EU. The designation provides incentives, including 10 years' of market exclusivity for the drug, if approved.
• The company believes there are ~112K patients with 22q in the EU and ~129K in Europe including the U.K.
• Zynerba noted that Zygel was previously granted orphan drug status in the EU for Fragile X syndrome (FXS) and in the U.S. for FXS and 22q. The drug also has a fast track designation in the U.S. to treat behavioral symptoms associated with FXS.
• ZYNE +4.85% to $0.66 premarket Nov. 15

For further details see: