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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
In the US, from January 1983 to June 2004, 249 orphan drugs received marketing authorization and 1,129 received different orphan drug designations, compared to fewer than ten such products in the decade prior to 1983. From 1983 until May 2010, the FDA approved 353 orphan drugs and granted orphan designations to 2,116 compounds.
In the US, from January 1983 to June 2004, a total of 1,129 different orphan drug designations have been granted by the Office of Orphan Products Development (OOPD) and 249 orphan drugs have received marketing authorization. In contrast, the decade prior to 1983 saw fewer than ten such products come to market.
Orphan drug status, which is given to drugs meant for the treatment of rare diseases, offers drug developers a path to faster approval and enhanced market exclusivity. So let's look at a small ...
Givinostat is in numerous phase II clinical trials (including for relapsed leukemias and myelomas), [15] and has been granted orphan drug designation in the European Union for the treatment of systemic juvenile idiopathic arthritis, [16] polycythaemia vera. [11] and Duchenne muscular dystrophy.
Maralixibat chloride, sold under the brand name Livmarli, is a medication used to treat cholestatic pruritus in people with Alagille syndrome. [5] [6] Maralixibat chloride is an ileal bile acid transporter (IBAT) inhibitor. [5] The most common side effects include diarrhea and abdominal pain (belly ache). [6]
NPS Pharmaceuticals is bringing its kids home. The biotech announced yesterday that it had reacquired the ex-North America rights to two drugs -- Revestive and Preotact -- that were licensed to ...
Amifampridine phosphate has orphan drug status in the EU for Lambert–Eaton myasthenic syndrome and Catalyst holds both an orphan designation and a breakthrough therapy designation in the US. In May 2019, the US Food and Drug Administration (FDA) approved amifampridine tablets under the brand name Ruzurgi for the treatment of Lambert-Eaton ...