Isis Pharmaceuticals antisense drug, mipomersen (Kynamro), recieved FDA approval for the treatment of a rare familal hypercholesterolemia (say fast 3x). The drug was developed in partnership with Genzyme (now part of Sanofi) and triggers a $25 M milestone payment to Isis.
This is the first time a drug of this type has been approved and Isis says that this validates its strategy and platform. Isis CEO, Stanley Crooke, said that the drug is "the culmination of two decades of work to create a new, more efficient drug technology platform." Isis says its pipeline shows the validity of the antisense approach which can be applied to a variety of diseases -- including rare or orphan ones. See Xconomy.
Posted by Bruce Lehr Jan 30th 2013.