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news.Isis Pharmaceuticals has reported that the FDA has granted orphan drug status to ISIS 333611 for the treatment of an inherited form of amyotrophic lateral sclerosis, commonly referred to as Lou Gehrig's disease.
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ISIS 333611 is a second-generation antisense drug in development for the treatment of amyotrophic lateral sclerosis (ALS) through the inhibition of Cu/Zn superoxide dismutase, a molecule that is associated with an inherited, aggressive form of ALS. It is Isis’s first drug treating neurodegenerative diseases to enter development and will be administered directly into the cerebral spinal fluid.
Frank Bennett, senior vice president of research at Isis, said: “Our preclinical success with ISIS 333611 demonstrates the applicability of antisense technology to treat a variety of diseases that affect the central nervous system.”