Common Sense for the Biochemist

Large Scale Biology announced today that the FDA had approved an orphan drug designation for a lysosomal acid lipase product that it was developing. This drug is being exclusively licensed to LSBC through a collaboration with researchers at the Cincinatti Children's Hospital. What does this mean? If this drug makes it through the FDA approval process, then LSBC would have seven years to exclusively market their drug for treatment of the disease that is targeted for. Orphan drugs are designed for treatment of diseases that affect less than 200,000 people in the U.S. This designation gives LSBC a little more leverage when negotiating with potential development and marketing partners for this drug. This is not the first orphan drug designation that LSBC has received. Their alpha-galactosidase treatment for Fabray's disease has also received the same designation. However, no developments regarding the progress of that product has been released recently.