Catalyst Pharma Fights Greed Charges Over An Orphan Drug

For the past month, shares in a small developer of novel drugs called Catalyst Pharmaceutical Partners have given up roughly half their value amid an unusual public relations war with a privately held drugmaker over a potentially lucrative market for an orphan medication. But the tale has some twists that include a decommissioned nuclear reactor and free dosing for existing patients. Here is the situation: Catalyst licensed a drug from Biomarin Pharmaceuticals called Firdapse to treatment Lambert-Eaton Myasthenic Syndrome, or LEMS, a rare autoimmune disorder that is characterized by muscle weakness and often associated with an underlying form of cancer. LEMS is considered a rare disease since only 3,000 or so people in the US are known to be affected. This means that Firdapse, which is in Phase III testing and will likely be submitted to the FDA for approval in early 2015, qualifies as an orphan drug. Already, there is speculation that Firdapse, which received breakthrough therapy designation from the FDA, may cost $60,000, which would yield at least $200 million in sales. However, Catalyst ceo Patrick McEnany tell us that a price has not yet been set. Seems straightforward, yes? There is a curve, though. A little-known, privately held company called Jacobus Pharmaceuticals has, for some 20 years, given a version of the drug called 3-4, Dap to approximately 200 patients for free under an FDA-approved compassionate use program. The drug is administered by individual doctors, m...
Source: Pharmalot - Category: Pharma Commentators Authors: Source Type: blogs