Press release
Anavex Announces U.S. FDA Orphan Drug Designation to ANAVEX®2-73 (blarcamesine) for the Treatment of Fragile X Syndrome
NEW YORK, Nov. 07, 2022 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company
About this update from Anavex Life Sciences Corp.
[{"type":"text","content":"NEW YORK, Nov. 07, 2022 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (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, today 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.1 At present, there is no approved treatment for Fragile X syndrome. “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.” 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 frequen...