Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) reported positive top line data from an open label exploratory Phase II FAB-C clinical trial assessing ZYN002 cannabidiol gel in pediatric and adolescent people with Fragile X syndrome. The study successfully achieved its primary endpoint, recording a 46% improvement in the total score of ADAMS at week twelve as against baseline. ZYN002 also recorded clinically meaningful improvements in distinct measures of the ABC-FXS, which address the major symptoms of FXS including temper tantrums, repetitive movements, hyperactivity and social avoidance.
Steven Siegel of Keck School of Medicine of USC, expressed that the results from the FAB-C study are extremely exciting and showcase that ZYN002 may have a deep effect on improving several disabling symptoms of Fragile X, like anxiety and difficult behaviors.
Fragile X is a tough genetic autism spectrum disorder, with intricate symptomatology that considerably impacts patients and their families. Several kids with Fragile X and their families combat with the lack of approved medications to safely cure their indications. This study indicates that ZYN002 is ready for the subsequent phase of development, and he consider that this drug shows great promise as a prospective treatment for these extremely difficult-to-treat symptoms.
With these results, Zynerba expects that it will meet with the U.S. FDA in 1H2018 with an objective of moving quickly into a major Phase 2/3 program in adolescent and pediatric patients with FXS in 2018. The U.S.FDA has allowed Zynerba Orphan Drug designation for the application of Cannabinoid as treatment of people with FXS.
Orphan Drug status is granted to novel medications that treat a rare condition or disease affecting fewer than 200,000 patients in the United States, and offers benefits including a 7-year period of United States marketing exclusivity upon marketing nod for the designated indication and may offer a fast path to market authorization.