Step Forward for Rx to Treat Rare Mental Condition

FDA grants orphan drug status to metadoxine in Fragile X Syndrome

/ Author:  / Reviewed by: Joseph V. Madia, MD Beth Bolt, RPh

The FDA has granted "Orphan Drug" status to a medication in the treatment of a rare disorder linked to autism.

Alcobra Ltd. (Nasdaq:ADHD) (the "Company"), an emerging biopharmaceutical company primarily focused on the development and commercialization of its proprietary drug candidate, MG01CI (Sustained-Release Metadoxine), to treat cognitive dysfunction, announced today that the U.S. Food & Drug Administration has granted "Orphan Drug" designation to Metadoxine in the treatment of Fragile X Syndrome. The Orphan Drug Act (ODA) provides for granting special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor. Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, as well as an extended period of market exclusivity following approval.

"We are pleased that the FDA has granted Orphan Drug status to Metadoxine, the active ingredient in MG01CI, in Fragile X," said Dr. Yaron Daniely, President and Chief Executive Officer of Alcobra Ltd. "We recently achieved positive results from a preclinical study of Fragile X and believe this outcome supports investigation in clinical trials which we plan to initiate in 2014. We recently raised approximately $38 million in a secondary offering, and we believe we now have enough cash to fund the Company through planned submissions of NDA and sNDA filings for MG01CI for adult ADHD, pediatric ADHD and Fragile X Syndrome."

In September this year, Alcobra announced positive findings from a pre-clinical study of Fragile X Syndrome. The study showed significant improvement in cognitive and social functioning following treatment with MG01CI in a valid animal model of Fragile X Syndrome (FMR1 knock-out mouse model). The study was funded in part by the FRAXA Research Foundation.

Fragile X syndrome (FXS) is a genetic condition that causes intellectual disability, behavioral and learning challenges and various physical characteristics. Behavioral characteristics can include ADHD, autism and autistic behaviors, social anxiety, stereotypic movements, poor eye contact, sensory disorders and increased risk for aggression. Fragile X Syndrome is the leading known genetic cause of autism, accounting for about 2-5% of cases. Fragile X represents an unmet medical need and a rare disease, as defined by the Orphan Drug Act. According to the U.S. Centers for Disease Control and Prevention (CDC), approximately one in 4,000 males and one in 8,000 females have Fragile X. The FDA has not approved any drugs specifically for the treatment of Fragile X or its symptoms.

Review Date: 
December 18, 2013