Mycophenolate mofetil
On June 6, 2006, the U.S. Food and Drug Administration (FDA) granted orphan drug status for mycophenolate mofetil (CellCept) in the treatment of pemphigus vulgaris, an autoimmune disorder which causes skin blistering. The efficacy of CellCept for treatment is being evaluated in a 52-week comparison study of 77 individuals, expected to be completed in 2007. CellCept, manufactured by Roche Laboratories, was previously approved as an anti-rejection drug for use with kidney, heart, and liver transplant recipients.
On June 6, 2006, the U.S. Food and Drug Administration (FDA) granted orphan drug status for mycophenolate mofetil (CellCept) in the treatment of pemphigus vulgaris, an autoimmune disorder which causes skin blistering. The efficacy of CellCept for treatment is being evaluated in a 52-week comparison study of 77 individuals, expected to be completed in 2007. CellCept, manufactured by Roche Laboratories, was previously approved as an anti-rejection drug for use with kidney, heart, and liver transplant recipients.
ISIS 301012
on May 30, 2006, the FDA approved orphan drug status for an investigational drug, ISIS 301012, for treatment of an inherited condition called homozygous familial hypercholesterolemia, which results in extremely high cholesterol. Isis Pharmaceuticals, the manufacturer, plans to rapidly develop the drug.
5-HMF
On June 6, 2006, the FDA approved orphan drug status for a 5-membered heterocyclic anti-sickling compound called 5-HMF for the treatment of sickle cell disease. Made by Xechem International, 5-HMF has specific interaction with sickle cells that does not affect other body proteins.
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