Breakthrough Designations Announced By Companies (Updated 9/5/2014):
o in combination with VX-809 in patients with two copies of the F508del mutation
o monotherapy for other CFTR mutations (Received FDA approval for eight additional mutations 2/21/2014)
o mantle cell lymphoma (Received FDA approval 11/13/2013 as Imbruvica)
o waldenstrom macroglobulinemia (WM)
o chronic lymphocytic leukemia (Received FDA approval 2/12/2014 as Imbruvica, expanded to additional CLL populations 7/28/2014)
Working with our partners in all sectors, Friends took ‘Breakthrough’ from concept, to scientific whitepaper, to bipartisan legislative solution, to a tool in full use by FDA to expedite the approval of multiple drugs in 13 months.
On Novemer 1, 2013, only a year after being signed into law, and 29 designations being announced, the first Breakthrough designation recieved full FDA Approval.
Here is a list of 2013's Breakthrough designations and approvals. Twelve of the year's 28 publicly announced designations were for cancer drugs, as well as two of its three Breakthrough approvals.
As knowledge about complex disease such as cancer and HIV/AIDS has expanded, researchers have been able to develop increasingly precise methods of treatment. Targeted therapies, a variety of drugs that targets specific molecular pathways, allow physicians and researchers to identify patients highly likely to respond to treatment. Frequently, these new drugs show major clinical activity and significant improvement over currently available treatment early in their development. In cases where new drugs show so much promise, and particularly in cases where existing treatment options are limited, the traditional multi-phase, sequential drug development process may not be appropriate.
Breakthrough Therapy (and Friends) in the News
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