Retrophin, a San Diego, CA based biopharmaceutical company announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation for its investigational drug, RE-024 for the treatment of pantothenate kinase-associated neurodegeneration (PKAN).  PKAN is a rare genetic neurodegenerative disease caused by homozygous or compound heterozygous mutation in the PANK2 (pantothenate kinase-2) gene, which encodes a enzyme that metabolizes vitamin B5 (pantothenate) to phosphopantothenate.  PKAN is characterized by progressive iron accumulation in the brain, resulting in parkinsonism, dystonia, dementia and ultimately death.  There is no treatment for this condition currently. RE-024 is a small molecule of phosphopantothenate replacement therapy developed by Retrophin and preclinical studies have demonstrated the ability of RE-024 to restore Coenzyme A, a metabolite involved in PANK2-mediated biochemical process.

 

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http://amda-1pla2o.client.shareholder.com/releasedetail.cfm?ReleaseID=911052