Alcobra: FDA Grants Orphan Drug Status To Metadoxine In Fragile X Syndrome

(RTTNews) - Alcobra Ltd. (ADHD), an emerging biopharmaceutical company primarily focused on the development and commercialization of its proprietary drug candidate, MG01CI or Sustained-Release Metadoxine, to treat cognitive dysfunction, announced Wednesday 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 or ODA provides for granting special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor.

"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," said Dr. Yaron Daniely, President and Chief Executive Officer of Alcobra Ltd.

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).

Fragile X syndrome or 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.