The landscape for rare disease treatments appears to be evolving rapidly, thanks to a confluence of regulatory momentum and a booming market forecast. Statements from key federal figures in recent months combined with market...more
On November 6th, 2017, the U.S. Food and Drug Administration (FDA) approved the drug Zelboraf (vemurafenib), for the treatment of Erdheim-Chester Disease (ECD). The FDA approved Zelboraf under Priority Review, Breakthrough...more
“Orphan drug” is a designation given to certain pharmaceutical and biological products (drugs) that would likely not be developed due to a relatively small patient population and limited potential for profitability. In the...more