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news.The US Food and Drug Administration’s (FDA) Office of Orphan Product Development (OOPD) has granted Orphan Drug Designation (ODD) to Anavex Life Sciences' ANAVEX 2-73 for the treatment of Rett syndrome.
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Anavex Life Sciences president and CEO Christopher U. Missling said: "The Orphan Drug Designation marks the first U.S. movement for ANAVEX 2-73. Rett syndrome is a devastating disease occurring in early childhood and almost exclusively in girls. As there is currently no cure and limited treatment, Rett syndrome represents a clear unmet clinical need.
"The acknowledgement from the Agency for this orphan designation allows us to formally move forward with the planned human clinical study in this disease indication, thereby potentially expanding the reach for ANAVEX 2-73 in addition to Alzheimer’s disease."
The FDA’s Office of Orphan Products Development advances the evaluation of drugs that show promise for the safe treatment of rare diseases, defined as those affecting fewer than 200,000 people in the United States. The designation provides sponsors with development and commercial incentives, including seven years of market exclusivity in the U.S., prioritized consultation by FDA on clinical studies and certain exemptions from or reductions in regulatory fees.
About Rett Syndrome
Rett syndrome is rare non-inherited genetic postnatal 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 even breathe easily.
The hallmark of Rett syndrome is near constant repetitive hand movements while awake. It 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, slowed brain and head growth, problems with walking, seizures, and intellectual disability.
There is currently no cure for Rett syndrome and treatment of the disorder is symptomatic. Management of the symptoms is done through a multidisciplinary approach utilizing medication for motor difficulties, breathing irregularities, and control of seizures through anticonvulsant drugs. Rett syndrome is caused by mutations in the MECP2 gene and strikes all racial and ethnic groups and occurs worldwide in approximately 1 in every 10,000-15,000 live female births.