Search results
Results from the WOW.Com Content Network
The 340B Drug Pricing Program is a US federal government program created in 1992 that requires drug manufacturers to provide outpatient drugs to eligible health care organizations and covered entities at significantly reduced prices. The intent of the program is to allow covered entities to "stretch scarce federal resources as far as possible ...
Targeting orphan drugs may sometimes fail. For example, Glybera was a $1 million injection used to treat a rare metabolic deficiency, but was removed from the market due to lack of demand. [82] However, there are many examples of orphan drug targeting proving to be a very profitable model for pharmaceutical companies. For example, Alexion ...
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
Since being signed into law 30 years ago, the Orphan Drug Act, or ODA, has resulted in the approval of more than 400 new drugs and biologic products in the United States. The goal of the ODA is to ...
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 ...
For premium support please call: 800-290-4726 more ways to reach us
Obecabtagene autoleucel, sold under the brand name Aucatzyl, is an anti-cancer medication used for the treatment of acute lymphoblastic leukemia. [1] [2] It is a CD19-directed genetically modified autologous T-cell immunotherapy.
Efficacy was evaluated in a single-arm cohort of an open-label, multicenter trial (SNDX-5613-0700, NCT04065399; AUGMENT-101) in 104 adult and pediatric participants (at least 30 days old) with relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene translocation. [3]