Search results
Results from the WOW.Com Content Network
This is especially true in the market for cancer drugs, as a 2011 study found that between 2004 and 2010 orphan drug trials were more likely to be smaller and less randomized than their non-orphan counterparts, but still had a higher FDA approval rate, with 15 orphan cancer drugs being approved, while only 12 non-orphan drugs were approved. [29]
Before Congress enacted the ODA in 1983 only 38 drugs were approved in the USA specifically to treat orphan diseases. [14] 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.
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 ...
bluebird bio, Inc., based in Somerville, Massachusetts, is a biotechnology company that develops gene therapies for severe genetic disorders. [1]The company's only - in the European Union (EU) - approved drug is betibeglogene autotemcel (Zynteglo), which treats transfusion-dependent beta thalassemia (TDT), a rare genetic blood disorder, and has been approved for use by the European Medicines ...
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
During his time leading the OOPD, Coté was "instrumental in implementing the Orphan Drug Act" [3] oversaw the approval of approximately 680 orphan designations. [4] Coté also developed a much stronger relationship between the FDA and the European Medicines Agency (EMA) by harmonizing communications and standards for orphan regulations. At the ...
Add yet another to the wave of orphan drugs preparing to hit the market. Pfizer (NYS: PFE) announced that the FDA has approved bosutinib as an orphan drug for chronic myelogenous leukemia, or CML.