Rare Daily Staff

The U.S. Food and Drug Administration granted Anavex Life Sciences Rare Pediatric Disease designation for ANAVEX 2-73, the company’s experimental therapy for Rett syndrome, a rare genetic neurological disorder.

Rett syndrome is a 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. 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. There is currently no cure for Rett syndrome.

ANAVEX 2-73 (blarcamesine) is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors and is in development to treat multiple neurological conditions including Alzheimer’s disease. The company said in preclinical studies, ANAVEX 2-73 exhibited anticonvulsant, anti-amnesic, neuroprotective, and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes ANAVEX 2-73 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Swedish Orphan Biovitrum sold its Priority Review voucher to AstraZeneca for $95 million.

“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,” said Christopher Missling, CEO of Anavex. “Our goal is to advance a potential treatment for Rett syndrome in order to bring medicines to patients as soon as possible.”


Photo: Christopher Missling, CEO of Anavex

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