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Home > News > FDA News > U.S. FDA Orphan Drug Designation to ANAVEX®2-73 (blarcamesine)

U.S. FDA Orphan Drug Designation to ANAVEX®2-73 (blarcamesine)

Author: Magicure Date: 2022-11-08

Nov. 07, 2022-- Anavex Life Sciences Corp. (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) disorders,  reports the U.S. Food and Drug Administration’s (FDA) has granted Orphan Drug Designation (ODD) to ANAVEX®2-73 (blarcamesine) for the treatment of Fragile X syndrome.

Fragile X syndrome (FXS) is the most common form of inherited intellectual disability and the most frequent single gene cause of autism spectrum disorder with an estimated population of approximately 62,500 in the US and 1,088,500 worldwide. At present, there is no approved treatment for Fragile X syndrome.

The FDA's Orphan Drug Designation program provides orphan status to drugs defined as those intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval.

Anavex Life Sciences’ product portfolio platform includes drug lead candidate ANAVEX®2-73 for the treatment of Alzheimer’s disease, Parkinson’s disease dementia, Parkinson’s disease and Rett syndrome and ANAVEX®3-71 for schizophrenia, frontotemporal dementia, and Alzheimer’s disease.

About Fragile X Syndrome and Autism Spectrum Disorder

Fragile X Syndrome is the most common form of inherited intellectual disability and the most frequent single gene cause of autism, affecting approximately 1 in 4,000 males and 1 in 6,000 females. The disorder is caused by the unstable expansion of a CGG repeat in the FMR1 gene that leads to abnormal methylation and suppression of FMR1 transcription with the resulting decrease in protein levels in the brain and other tissues. The average age of Fragile X syndrome diagnosis for boys and girls are 35 to 37 months and 42 months, respectively. Behavioral abnormalities, including autism spectrum disorder, are common.

Autism spectrum disorder is a behavioral diagnosis while Fragile X syndrome is a medical/genetic diagnosis. Many studies have evaluated the link between Fragile X syndrome and autism spectrum disorder over the last few decades. Since many children with Fragile X syndrome are interested in social interactions, they may not meet the diagnostic criteria for autism spectrum disorder, even though they exhibit some features such as poor eye contact, shyness, social anxiety, hand-flapping and sensory issues. Autism is much more common in boys than in girls with Fragile X syndrome. According to the CDC, a national parent survey found that 46% of males and 16% of females with Fragile X syndrome have been diagnosed or treated for autism spectrum disorder.

“The Orphan Drug Designation highlights the potential to expand the therapeutic profile of ANAVEX®2-73 into the largest portion of autism spectrum disorder, Fragile X syndrome,” said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. “We look forward to working with the Fragile X syndrome community to rapidly advance ANAVEX®2-73 as a potential treatment for Fragile X syndrome while we continue to expand late-stage clinical investigation of ANAVEX®2-73 as part of its precision medicine platform technology for both neurodevelopmental and neurodegenerative indications.”

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