2011 US FDA orphan drug designations

In Uncategorized by Pinky Fadullon

orphan drug Pharmaceutical companies turn to orphan drug development, a viable alternative to what one quipped as the death of blockbuster drugs.

Applications for orphan drug designations have been climbing nationwide in recent years. In 2010, the U.S. Food and Drug Administration’s Office of Orphan Products Development received 323 applications and approved 14 orphan drugs. In 2009, there were 250 applications and 17 approvals. Triangle Business Journal, July 22, 2011

In 2011’s first half alone, this is what it’s looking like:

· Orphan drug designation to 4SC cancer

· Immune System Therapeutics Limited (IST), announced that its breakthrough antibody treatment for terminal blood cancer had received Orphan Drug Designation

· Provectus Pharmaceuticals received orphan drug designation for Rose Bengal, the active ingredient in its novel oncology drug PV-10, for the treatment of hepatocellular carcinoma, the most common form of primary liver cancer

· Tengion for its Neo-Urinary Conduit, for treatment of bladder dysfunction requiring incontinent urinary diversion

· Bayer HealthCare Pharmaceuticals investigational compound regorafenib has been granted orphan drug status for the treatment of patients with gastrointestinal stromal tumors

· CytRx Corporation’s INNO-206 received orphan drug designation for the treatment of patients with soft tissue sarcomas

What’s in your orphan drug pipeline?

To learn more about this rising sector and meet industry movers and shakers from pharmas to biotechs, to FDA to payers to patient groups, join the 2nd Annual World Orphan Drug Congress USA.