FDA grants orphan status to Raptor's Huntington's drug


NOVATO, Calif. Raptor Pharmaceuticals announced Monday that the Food and Drug Administration had granted orphan drug designation to a drug with potential to treat Huntington’s disease, a rare hereditary disease that leads to neurological deterioration and death.

The FDA has approved the drug, cysteamine bitartrate, for the treatment of the lysosomal storage disease nephropathic cystinosis. Preclinical findings have shown that cysteamine increases levels of the neurotrophic factor BDNF in the brains of mice. Levels of BDNF, which protects brain cells, may indicate efficacy of cysteamine in treating Huntington’s disease. Raptor will begin Phase II testing later this year.

The 1983 Orphan Drug Act encourages companies to develop therapies for rare and serious diseases that affect 200,000 or fewer people in the U.S. FDA orphan drug designation streamlines the regulatory process and grants seven years’ market exclusivity after a drug application receives approval.

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