RARE Daily

Origin’s Rapid Path to Seek Approval for Treatment of Ultra-Rare Metabolic Condition

December 20, 2019

Earlier this month, Origin Biosciences initiated a rolling submission with the U.S. Food and Drug Administration for approval to market an experimental therapy to treat the ultra-rare metabolic condition molybdenum cofactor deficiency (MoCD) Type A. The condition manifests itself shortly after birth and leads to difficulty feeding and intractable seizures. Children with the condition have a median survival of three years and those who live longer suffer irreversible damage to their central nervous system. The rolling submission comes about a year and a half after BridgeBio acquired the therapy from Alexion Pharmaceuticals and formed Origin Biosciences to develop it. We spoke to Neil Kirby, CEO of Origin, about MoCD, how its synthetic enzyme co-factor works to treat it, and the plans for Origin beyond the submission for approval.ultra-rare diseaseMoCDmolybdenum cofactor deficiencydrug developmentbusiness models

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