Transdermal Cannabidiol Gets Orphan Drug Status for 22q11.2 Deletion Syndrome

The efficacy and safety of Zygel is being evaluated in the INSPIRE study.

The Food and Drug Administration (FDA) has granted Orphan Drug designation to Zygel™ (cannabidiol gel; Zynerba Pharmaceuticals) for the treatment of 22q11.2 deletion syndrome (22q), a chromosomal disorder that results in physical abnormalities (ie, palate abnormalities, heart defects, gastrointestinal [GI] issues) and neuropsychiatric and behavioral symptoms (ie, anxiety, withdrawn behavior, social interaction problems).

Zygel is a pharmaceutically-manufactured cannabidiol (CBD) formulated as a permeation-enhanced transdermal gel. It is designed to provide controlled drug delivery into the bloodstream through the skin with a lower incidence of GI side effects. The investigational therapy is believed to be beneficial in addressing the behavioral symptoms associated with 22q.

The efficacy and safety of Zygel is being evaluated in the INSPIRE study, a 14-week, open-label, multidose phase 2 trial.  Patients aged 6 to 17 years with genetically-confirmed 22q (n=20) will be administered weight-based doses of Zygel, which is applied every 12 hours to the upper arms and shoulders. Efficacy will be assessed using the Aberrant Behavior Checklist-Community (ABC-C); the Anxiety, Depression and Mood Scale (ADAMS); the Columbia Suicide Severity Rating scale (C-SSRS); the Qualitative Caregiver Reported Behavioral Problem Survey; and Clinical Global Impression – Severity and Improvement scale.

“Zynerba is committed to developing Zygel CBD gel in certain rare and near-rare conditions, including 22q, for which there is an urgent need for new, innovative therapeutics,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “The receipt of this designation represents another important milestone for us, and we look forward to working closely with the FDA to develop Zygel in pediatric and adolescent patients with 22q as expeditiously as possible.”

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The Company is also investigating Zygel for the treatment of behavioral symptoms associated with Fragile X syndrome and autism spectrum disorder.

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Zynerba Pharmaceuticals receives Orphan Drug designation for cannabidiol for the treatment of 22q11.2 deletion syndrome. Accessed September 17, 2020.