Tuesday, September 23, 2014

ISIS 333611, Orphan Drug for ALS, Approved 2007

Seven years before major ALS headlines, spurred on by the Ice Bucket Challenge, the FDA approved a curious drug with a curious patent # 333611, for ALS.  Notice the 333 and 611.
Isis Pharmaceuticals, Inc. (Nasdaq: ISIS) announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISIS 333611 for the treatment of an inherited form of amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig's disease. ALS is a progressive neurodegenerative disease resulting in the loss of muscle control that can lead to death. ISIS 333611, a second-generation antisense drug, is Isis' first drug treating neurodegenerative diseases to enter development and will be administered directly into the cerebral spinal fluid.
"ALS is a terrible disease in need of innovative and efficacious new therapies. We are pleased that ISIS 333611 has been granted orphan drug status for this small but important patient population. In our preclinical studies, we demonstrated that direct delivery of an antisense drug onto the spinal fluid via an implantable pump effectively reduced SOD1, a protein associated with the inherited form of ALS," said C. Frank Bennett, Ph.D., Senior Vice President of Research at Isis Pharmaceuticals, Inc. "Our preclinical success with ISIS 333611 demonstrates the applicability of antisense technology to treat a variety of diseases that affect the central nervous system. In addition, with funding from CHDI, we recently initiated a separate drug discovery program in Huntington's Disease, another fatal neurodegenerative disease."
Archived Article from 2007:  http://ir.isispharm.com/phoenix.zhtml?c=222170&p=irol-newsArticle_pf&ID=1289508&highlight=

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