In Brief: Orphan Medical's Cystadane
Executive Summary
Orphan Medical's Cystadane: FDA approves betaine anhydrous oral solution Oct. 25 for "the treatment of homocystinuria to decrease elevated homocysteine blood levels," agency approval letter states. Included within the category of homocystinuria are deficiencies or defects in cystathionine beta synthase (CBS); 5,10-methylenetetrahydrofolate reductase (MTHFR); and cobalamin cofactor metabolism (cbl). Cystadane is classified as a "1PV" drug, denoting a new molecular entity with an orphan designation receiving a priority review...
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