This, for the drug Esbriet (pirfenidone), which is expected to generate $1B in yearly revenue by 2019 for the treatment of idiopathic pulmonary fibrosis (IPF), an orphan disease. On February 28, 2011, the European Commission (EC) granted marketing authorization in all 28 EU member states. It is also available in Canada, Japan, South Korea, China, India, Argentina and Mexico, BUT NOT IN THE U.S., in spite of the drug receiving FDA Orphan Designation, Priority Review Designation, Fast Track Status and Breakthrough Designation. What happened? In one of two p3 trials completed in 2009, the primary endpoint was not met (p=0.501). For this failure, the company was set back by 5 years by the FDA. A third p3 trial was mandated and now they are resubmitting their NDA to the FDA in 3rd qtr 2014.
Does all this sound familiar? Orphan drug. Estimated revenue= $1B/yr. Relaxed standards abroad. BS FDA designations mean nothing in the US if you fail primary endpoint. If we follow the pattern, look for approval in the EU and possibly Japan. We shall see.
THX B-L: I guess UBS knew what they were talking about last January. They listed IDIX and ITMN as buyout candidates this year. Insmed is on that list and UBS has not wavered!
"The UBS report also lists five stocks that pending successful trial outcome and data presentations this year could become strong takeover candidates as well. They included Idenix Pharmaceuticals Inc. (NASDAQ: IDIX), Insmed Inc. (NASDAQ: INSM), InterMune Inc. (NASDAQ: ITMN), Puma Biotechnology Inc. (NYSE: PBYI) and Vertex Pharmaceuticals Inc. (NASDAQ: VRTX). All the stocks are Buy rated at UBS and could jump much higher with a takeover bid."