New FDA Orphan Drugs: CDX-110, ATryn, ISIS 333611

Jill Taylor

December 21, 2007

December 21, 2007 -- The US Food and Drug Administration (FDA) has granted orphan drug designation to CDX-110 for the treatment of glioblastoma multiforme, ATryn for the treatment of hereditary antithrombin deficiency, and ISIS 333611 for the treatment of amyotrophic lateral sclerosis.

Orphan Drug CDX-110 for Glioblastoma Multiforme

On December 7, Celldex Therapeutics announced that the FDA granted orphan drug designation to CDX-110 for the treatment of glioblastoma multiforme (GBM), an aggressive, high-grade malignancy of the brain that kills most patients within a year.

Because of the diffuse nature of the disease, GBM is exceedingly difficult to treat. The standard of care -- surgical resection of the tumor followed by radiotherapy and chemotherapy -- has essentially remained unchanged for decades and may only extend survival by months.

CDX-110 is a tumor immunotherapy that targets a common form of mutant epidermal growth factor receptor, EGFRvIII, which is implicated in several forms of cancer, including glioblastoma. EGFRvIII has minimal expression in normal tissues and is thus considered a target of interest in immunotherapy.

Orphan Drug ATryn for Hereditary Antithrombin Deficiency

On December 7, the FDA granted orphan drug designation to ATryn (GTC Biotherapeutics, Inc) for the treatment of hereditary antithrombin deficiency (HD), a genetic disorder that increases the risk for blood clots.

HD is estimated to occur in 1 of every 500 to 5000 people in the general population. In HD, the deficiency in antithrombin, a protein in blood that functions as a mild blood thinner, raises the risk of dangerous conditions caused by clots, including deep vein thrombosis, pulmonary embolism, Budd-Chiari syndrome, or sinus vein thrombosis.

ATryn is a recombinant form of human antithrombin. The drug has previously been approved by the European Union for use in HD patients undergoing surgery.

Orphan Drug ISIS 333611 for Amyotrophic Lateral Sclerosis

On December 13, Isis Pharmaceuticals, Inc, announced that the FDA granted orphan drug designation to ISIS 333611for the treatment of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease that affects motor neurons of the central nervous system.

Also known as Lou Gehrig's disease, ALS is characterized by loss of voluntary muscle action and muscle atrophy. Early symptoms of ALS include weakness of the arms and the legs; as the disease progresses, patients gradually lose the ability to initiate and control muscle movement and, in later stages, may become completely paralyzed.

ISIS 333611 is a second-generation antisense drug that inhibits Cu/Zn superoxide dismutase (SOD) and antioxidant enzyme that defends the cell against damage by oxygen free radicals. Mutations of the SOD1 gene that encode Cu/Zn SOD convert the protective enzyme into a pro-oxidant form that is destructive to motor neurons and have been found to occur in familial ALS.

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