Orphan Medical's Cystadane for homocystinuria is firm's second approval within month.
ORPHAN MEDICAL CYSTADANE IS SECOND APPROVAL IN ONE MONTH for the Minnetonka, Minn.-based pharmaceutical company. Betaine anhydrous oral solution was approved Oct. 20 for "the treatment of homocystinuria to decrease elevated homocysteine blood levels" ("The Pink Sheet" Oct. 28, In Brief). Cystadane is the first drug to receive FDA approval for the treatment of homocystinuria, a rare genetic disorder caused by various inborn errors of methionine metabolism that affects about 1,000 patients in the U.S. The drug was classified a "1PV" by FDA, denoting a new molecular entity with orphan status given a priority review.
You may also be interested in...
Newly released Medicare Part D data sheds light on the sales hit that branded pharmaceutical manufacturers will face when the coverage gap discount program gets under way in 2011
FDA appears headed for a showdown with clinicians and the pharmaceutical industry over the proposed new clinical trial endpoints for acute bacterial skin and skin structure infections, the guidance's approach for justifying a non-inferiority margin and proposed changes in the types of patients that should be enrolled in trials
Specialty drug maker Shire has quietly begun scouting deals with a brand-new $50 million venture fund, the latest of several in-house investment arms to launch with their parent company's pipelines, not profits, as the measure of their worth