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The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy that depends on the legislation (if there is any) of the country. Designation of a drug as an orphan drug has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and ...
In contrast, the decade prior to 1983 saw fewer than ten such products come to market. From the passage of the ODA in 1983 until May 2010, the FDA approved 353 orphan drugs and granted orphan designations to 2,116 compounds. As of 2010, 200 of the roughly 7,000 officially designated orphan diseases have become treatable. [13]
Lists of all drugs that have received orphan status in the United States and Europe are available from the U.S. Food and Drug Administration and the European Commission, respectively: FDA List of Orphan Designations and Approvals [dead link ] European Commission Register of designated Orphan Medicinal Products
The FDA grants Orphan Drug and Rare Pediatric Disease designations to Ultragenyx (RARE) and partner GeneTx Biotherapeutics' GTX-102 for the treatment of Angelman Syndrome.
The FDA bestows an orphan drug designation on Reata Pharmaceuticals' (RETA) candidate, bardoxolone, for treating patients with autosomal dominant polycystic kidney disease.
The FDA bestows an Orphan Drug status to aTyr Pharma's (LIFE) lead candidate, efzofitimod, for the treatment of systemic sclerosis. Shares rise.
In 2010, the FDA granted orphan drug status to tasimelteon, then regarded as an investigational medication, for use in totally blind adults with N24HSWD. [14] (Through mechanisms such as easing the approval process and extending exclusivity periods, orphan drug status encourages development of drugs for rare conditions that otherwise might lack ...
The FDA grants orphan drug designation to Arcturus Therapeutics' (ARCT) candidate, ARCT-810 for treating patients with ornithine transcarbamylase deficiency, the most common urea cycle disorder.