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Zynerba Pharmaceuticals Receives Fast Track Designation for Zygel™ for the Treatment of Behavioral Symptoms Associated with Fragile X Syndrome (FXS)
- Designation Facilitates and
- Enrollment Progressing in Pivotal CONNECT-FX Trial of Zygel in FXS, with Data Expected in the Second Half of 2019 -
“The FDA’s decision to grant Fast Track Designation for Zygel underscores the significance and severity of the unmet medical need that exists for patients living with Fragile X Syndrome and their caregivers,” said
Zynerba is conducting a pivotal trial to assess the efficacy and safety of Zygel as treatment for the behavioral symptoms of FXS in pediatric and adolescent patients (three through 17 years of age). Zygel is a pharmaceutically-manufactured CBD formulated as a patent-protected permeation-enhanced clear gel, designed to provide controlled transdermal drug delivery into the bloodstream.
About Fast Track Designation
Fast Track Designation is intended to facilitate development and expedite review of drugs to treat serious or life-threatening conditions so that a product can reach the market expeditiously. A drug that is intended to treat a serious or life-threatening condition that demonstrates the potential to address an unmet medical need may qualify for Fast Track designation. Features of this designation include opportunities for frequent interactions with the review team. These include meetings with the
About Fragile X Syndrome (FXS)
Fragile X syndrome is a rare genetic developmental disability that is the leading known cause of both inherited intellectual disability and autism spectrum disorder, affecting 1 in 3,600 to 4,000 males and 1 in 4,000 to 6,000 females. It is the most common inherited intellectual disability in males and a significant cause of intellectual disability in females. FXS is caused by a mutation in the Fragile X Mental Retardation gene (FMR1) located on the X chromosome and leads to dysregulation of the endocannabinoid pathway including the reduction in endogenous cannabinoids (2-AG and anandamide). The disorder negatively affects synaptic function, plasticity and neuronal connections, and results in a spectrum of intellectual disabilities and behavioral symptoms, such as social avoidance and irritability. In the US, there are about 71,000 patients suffering with FXS.
Zygel (CBD gel) is the first and only pharmaceutically-manufactured CBD 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 FXS and other neuropsychiatric conditions may be associated with a disruption in the endocannabinoid (EC) system. Clinical and anecdotal data suggest that CBD may modulate the EC system and improve certain core social and behavioral symptoms, including social avoidance (prefers isolation from others, prefers solitary activities, avoids new social activities), irritability (aggressive to others, tantrums/outbursts, and stubbornness), and social unresponsiveness/lethargy (lack of attention/interaction, inactive/lack of movement and can resist physical contact).
Enrollment is ongoing in the multi-national, randomized, double blind placebo controlled Clinical study of Cannabidiol (CBD) in Children and Adolescents with Fragile X (CONNECT-FX), a pivotal clinical trial of ZYN002 in FXS (https://www.connectfxtrial.com/); topline data from CONNECT-FX are expected in the second half of 2019. Additionally, Zynerba expects topline data from its Phase 2 Open Label Study to Assess the Safety and Efficacy of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Developmental and Epileptic Encephalopathy (BELIEVE 1) clinical trial in the third quarter of 2019. Zynerba has also initiated a Phase 2 study of Zygel in Autism Spectrum Disorder, with data expected in the first half of 2020.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from the Company’s current expectations. For example, there can be no guarantee that the Company will obtain approval for Zygel from the
Source: Zynerba Pharmaceuticals, Inc.