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MADISON, Wis. — The Food and Drug Administration has granted orphan drug designation to a treatment made by Mithridion for a rare neurological disorder, Mithridion said Monday.
The FDA gave the designation to MCD-386CR, an investigational drug for progressive supranuclear palsy, or PSP, which recently completed early-stage clinical trials. PSP is a progressive brain disease in which neurons in the regions of the brain that control eye movements, balance, walking, speech and cognition degenerate, leading to slowed thinking and difficulties with reasoning, planning and shifting between tasks.
“Orphan status will help us immeasurably to bring together the resources and support needed to evaluate MCD-386CR in this rare but important disease and indeed other devastating brain diseases for which there are no current therapies,” Mithridion CEO Trevor Twose said. “Based on results in preclinical studies, we believe MCD0386CR potentially will help restore cognition, so vital to human functioning, and potentially could treat the underlying processes causing the degeneration of neurons in PSP.”
The FDA gives orphan drug designation to investigational treatments for diseases that affect fewer than 200,000 people in the United States.