FDA grants fast track designation to metadoxine for fragile X syndrome

Alcobra recently announced that the FDA has granted fast track designation to metadoxine for the treatment of fragile X syndrome, a genetic condition that causes a range of developmental problems including learning disabilities and cognitive impairment.

Also known as Martin-Bell syndrome, fragile X syndrome is the most common cause of inherited mental retardation, intellectual disability and autism, affecting approximately 1 in 4,000 males and 1 in 8,000 females in the United States. Currently, there are no approved therapies for fragile X syndrome.

The metadoxine extended release (MDX) formulation — developed as a potential treatment for attention-deficit/hyperactivity disorder and fragile X syndrome — is not a stimulant, but instead acts as a monoamine-independent modulator of gamma-aminobutyric acid transmission.

The FDA based its decision on preclinical studies that demonstrated that metadoxine was generally well tolerated with no identified safety concerns, and has no potential for abuse or addiction. In addition, MDX is currently in phase 2 development for both pediatric ADHD and fragile X syndrome.

“We are pleased that the FDA has recognized the potential of MDX in fragile X syndrome and granted fast track designation for this indication,” Yaron Daniely, PhD, president and CEO of Alcobra said in a press release. “We look forward to our upcoming meeting with the FDA to determine next steps in advancing the development program for MDX in this area of serious unmet medical need.”

The FDA provides fast track status to facilitate the development of new products for serious or life-threatening conditions that demonstrate the potential to address unmet medical needs, with the goal of getting important new products to patients earlier. Fast track status also will allow the company to work closely with the FDA to expedite the review of aspects of the pharmaceutical MDX program to improve the efficiency of product development.