15 Nov 22
Zynerba Pharmaceuticals Announces European Commission Has Granted Orphan Drug Designation for Zygel™ in 22q11.2 Deletion Syndrome
Devon, PA, November 15, 2022 — Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders, today 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).
“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, Chairman and Chief Executive Officer of Zynerba. “Orphan drug designation is another important step forward in advancing Zygel as a potential new treatment option for patients with 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.
Zygel is the first and only pharmaceutically-manufactured cannabidiol formulated as a patent-protected permeation-enhanced clear gel, designed to provide controlled drug delivery into the bloodstream transdermally (i.e. through the skin). Recent studies suggest that cannabidiol may modulate the endocannabinoid system and improve certain behavioral symptoms associated with neuropsychiatric conditions. Zygel is an investigational drug product in development for the potential treatment of behavioral symptoms associated with Fragile X syndrome (FXS), 22q11.2 deletion syndrome (22q) and autism spectrum disorder (ASD). The Company has received orphan drug designation for cannabidiol, the active ingredient in Zygel, from the FDA and the European Commission in the treatment of FXS and the treatment of 22q. Additionally, Zygel has been designated a Fast Track development program for treatment of behavioral symptoms of FXS.
About 22q11.2 Deletion Syndrome (22q)
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 esophageal / GI issues, as well as debilitating neuropsychiatric and behavioral 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 behaviors such as self-care and communication skills that affect daily life. Children with 22q also experience withdrawn behavior, 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 83,000 people living with 22q in the U.S. In addition, Zynerba believes that there are approximately 112,000 patients with 22q in the EU and approximately 129,000 in Europe if the United Kingdom is included.
About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X syndrome, 22q11.2 deletion syndrome and autism spectrum disorder. Learn more at www.zynerba.com and follow us on Twitter at @ZynerbaPharma.
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