PRAX - Praxis Precision Medicines' PRAX-562 treatment wins FDA 'orphan drug' status

Praxis Precision Medicines (PRAX) announces that the U.S. FDA has granted orphan drug designation for PRAX-562 for the treatment of SCN8A-DEE, a rare developmental and epileptic encephalopathy caused by a variant in the SCN8A gene.The FDA’s orphan drug designation program is designed to encourage and facilitate the development of investigational treatments for rare diseases that affect fewer than 200,000 people in the United States.The designation provides various development and commercial incentives, including tax credits for clinical research costs, waiver or partial payment of application fees and market exclusivity for seven years following FDA approval.PRAX-562 is a small molecule and is the first selective persistent sodium current blocker in development for the treatment of a wide range of rare central nervous system disorders.Patients with SCN8A-DEE have significant cognitive disabilities, ranging from moderate to severe; often movement disorders, such as dystonia or ataxia; and problems in other body systems such as gastrointestinal or ocular.

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Praxis Precision Medicines' PRAX-562 treatment wins FDA 'orphan drug' status