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Anavex Life Sciences Receives Rare Pediatric Disease Designation from FDA for ANAVEX®2-73 (blarcamesine) for the Treatment of Rett Syndrome
NEW YORK, Nov. 14, 2019 (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. 14, 2019 (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) diseases, today announced that it received the Rare Pediatric Disease (RPD) designation from the U.S. Food and Drug Administration (FDA) for ANAVEX®2-73 (blarcamesine) for the treatment of Rett syndrome, a rare genetic neurological disorder associated with severe physical and cognitive impairments that afflicts mostly girls.\n The RPD designation provides priority review by the FDA to encourage treatments for rare pediatric diseases. Under the RPD program, a sponsor who receives an approval for a drug for a \"rare pediatric disease\" may qualify for a voucher that can be redeemed to receive a priority review by the FDA of a subsequent marketing application for a different product. “We are pleased to have received the rare pediatric designation from the U.S. FDA for ANAVEX®2-73 (blarcamesine) for young patients with Rett syndrome,” said Christopher U. Missling, Ph.D., Chief Executive Officer of Anavex. “This designation for ANAVEX®2-73 (blarcamesine) underscores the significant unmet medical need for patients with this genetic disease affecting nearly every aspect of the child’s life: their ability to speak, walk, eat and easily breathe. Our goal is to advance a potential treatment for Rett syndrome in order to bring medicines to patients as soon as possible.” About Rett Syndrome Rett syndrome is a devastating, non-inherited genetic post-natal progressive neurodevelopmental disorder that occurs almost exclusively in girls and leads to severe impairments, affecting nearly every aspect of the child’s life: their ability to speak, walk, eat and easily breathe. The hallmark of Rett syndrome is near constant repetitive hand movements while awake. The disease is characterized by normal early growth and development (6 to 18 months) followed by a slowing of development, loss of purposeful use of the hands, distinctive hand movements, autistic features, slowed brain and head growth, ataxia, seizures and intellectual disability. Rett syndrome is caused by mutations in the ...