GEXVal Receives FDA Orphan Drug Designation for GXV-001 in Fragile X Syndrome

株式会社GEXVal
November 19, 2025

GEXVal Receives FDA Orphan Drug Designation for GXV-001 in Fragile X Syndrome

GEXVal Inc. (President & CEO: Juran Kato, Headquarters: Fujisawa, Kanagawa, Japan; hereinafter "GEXVal") announces that GXV-001, its drug candidate being studied for Fragile X syndrome¹⁾ (FXS), has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA).

The FDA's Orphan Drug Designation database is publicly accessible, allowing anyone to search and verify designation status. (Search URL: https://www.accessdata.fda.gov/scripts/opdlisting/oopd)

About Orphan Drug Designation (ODD)
The ODD program was established by the FDA to encourage the research and development of treatments for rare diseases affecting fewer than 200,000 patients in the United States. Applications are submitted to the FDA's Office of Orphan Products Development (OOPD) and evaluated based on data demonstrating a scientifically plausible basis for development in the target rare disease.

Companies that receive ODD are eligible for the following benefits and incentives:
Waiver of FDA application fees, reducing the financial burden of development. A federal tax credit for a portion of qualified clinical trial expenses. Enhanced access to scientific guidance from the FDA, enabling closer collaboration with regulators throughout the development process. Upon approval, up to seven years of market exclusivity, providing a foundation for sustained investment in rare disease development.
It is important to note that ODD is a designation intended to support investigational research and development. It does not constitute FDA approval of the safety or efficacy of any medicinal product.

Next Steps
With support from FRAXA Research Foundation²⁾ and the Japan Agency for Medical Research and Development (AMED)³⁾, GEXVal is advancing preparations for a Phase 2a clinical trial of GXV-001. The receipt of FDA ODD reflects the body of preclinical evidence generated through the FRAXA Drug Validation Initiative (FRAXA-DVI) program, and represents an important foundation for the next stage of development.
GEXVal remains committed to reaching patients and families living with FXS with new options as soon as possible, and will continue to advance its clinical development program while pursuing the necessary funding to support this work.

1) About Fragile X syndrome (FXS): Fragile X syndrome is a rare genetic condition associated with features of  autism spectrum disorder. According to FRAXA Research Foundation, it is the most common genetic cause of autism and intellectual disabilities.

2) About FRAXA Research Foundation (FRAXA): FRAXA is a nonprofit organization headquartered in Massachusetts, USA, dedicated to supporting research and raising awareness for the development of treatments for Fragile X Syndrome.
For more information, visit https://www.fraxa.org

3) About Japan Agency for Medical Research and Development (AMED): AMED is an agency that supports research and development necessary for medical advancement, providing comprehensive support from basic research to practical application in the development of new treatments and medicines.
For more information, visit https://www.amed.go.jp/en/aboutus/index.html

[About GEXVal]
GEXVal strives to create and develop innovative pharmaceuticals for unmet medical needs, ensuring Treatment Reaches the Unreached with focus on rare diseases and underserved medical conditions. By leveraging our proprietary AI-powered pharmacoinformatics technology, we illuminate paths to breakthrough therapies, identifying hidden potential in drug candidates to deliver life-changing medicines that bring new hope to patients and their families.

有关本事宜请咨询:
Head of Corporate Office
Atsushi Sugizaki
info@gexval.com