Zynerba Pharmaceuticals, Inc., the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, announced that the U.S. Food and Drug Administration has granted orphan drug designation for cannabidiol (CBD) for use in treating 22q11.2 deletion syndrome (22q). 22q is a rare midline condition featuring physical abnormalities and debilitating neuropsychiatric and behavioural symptoms including anxiety, withdrawn behaviour, and social interaction problems.

“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. “We are pleased that the FDA shares our sense of urgency regarding the development of effective therapeutics in this important patient population. 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.”

Under the Orphan Drug Act (ODA), the FDA may grant orphan drug designation to drugs intended to treat rare diseases or conditions that affect fewer than 200,000 individuals in the U.S. The first NDA applicant to receive FDA approval for a particular active moiety to treat a particular disease with FDA orphan drug designation is entitled to various incentives of the ODA, including tax credits for qualified clinical testing, waiver of new drug application (NDA) / biologics license application (BLA) user fees, and eligibility for a seven-year exclusive marketing period for that drug and use upon marketing approval.

As the second most common chromosomal disorder after Down syndrome, 22q is caused by a small missing piece of the 22nd chromosome. The deletion occurs near the middle of the chromosome at a location designated q11.2. It is considered a mid-line condition, with physical symptoms including characteristic palate abnormalities, heart defects, immune dysfunction, and oesophagal / GI issues, as well as debilitating neuropsychiatric and behavioural challenges. Anxiety is among the most common neuropsychiatric symptoms of 22q and researchers have found that for children with 22q, anxiety is linked to poorer adaptive behaviours such as self-care and communication skills that affect daily life. Children with 22q also experience withdrawn behaviour, ADHD, cognitive impairment, and autism spectrum disorder that affect communication and social interaction. Later in life, they are at an increased risk of developing mental illnesses such as schizophrenia. It is estimated that 22q occurs in between one in 3,000 and one in 6,000 live births, suggesting that there are approximately 81,000 people living with 22q in the U.S.