Anavex Life Sciences Receives Positive Opinion for Orphan Designation from the European Medicines Agency for ANAVEX®2-73 for the Treatment of Rett Syndrome
GlobeNewswireJuly 29, 2019, 1:00 PM GMT+2
NEW YORK, July 29, 2019 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (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 the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on Anavex’ application for orphan designation of ANAVEX®2-73 for the treatment of Rett syndrome. The positive opinion issued by COMP will be sent to the European Commission, which is expected to grant the orphan designation within 30 days.
Under the EMA’s Regulation (EC) No. 141/2000 an orphan medicinal product designation gives companies access to reduced regulatory fees, protocol assistance and guidance on preparing a dossier that will meet European regulatory requirements and thereby maximize the chance of success at the time of marketing authorization. Once approved, an orphan drug is also granted 10 years of market exclusivity in the European Union (EU), hence protecting it from competition from similar medicines, which cannot be marketed during this 10-year exclusivity period for this indication.
The EMA grants orphan medicinal product designation based upon several criteria: the life threatening and debilitating nature of the condition; the medical plausibility of the proposed orphan indication; a prevalence in Europe of less than 5 cases for each 10,000 of population; no satisfactory method of diagnosis, prevention or treatment exists or if such method exists the medicinal product will be of significant benefit to those affected by that condition.
ANAVEX®2-73 had previously received orphan drug designation from the United States (U.S.) Food and Drug Administration (FDA) for the treatment of Rett syndrome.
“ANAVEX®2-73 has the potential to provide patients and physicians with a much-needed treatment option for Rett syndrome, a rare genetic disorder. The COMP’s adoption of a positive opinion for ANAVEX®2-73 orphan drug designation is another important milestone for this program, which continues to advance rapidly,” said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. “In addition to Rett syndrome1, Anavex has ongoing clinical development programs for ANAVEX®2-73 for the treatment of Alzheimer’s disease2 and Parkinson’s disease dementia3.”
